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# KrabbeConnect

Connecting Patients and Research

## Sitemaps

- [XML Sitemap](https://krabbeconnect.org/sitemap.xml): Contains all public & indexable URLs for this website.

## Posts

- [KrabbeConnect Secures Generous Funding from the Rosenau Family Research Foundation for Groundbreaking Krabbe Disease Study](https://krabbeconnect.org/2023/11/krabbeconnect-secures-generous-funding-from-the-rosenau-family-research-foundation-for-groundbreaking-krabbe-disease-study/) - Minneapolis, MN, November 30th, 2023 — KrabbeConnect, a pioneering organization dedicated to supporting individuals and families affected by Krabbe disease, is proud to announce the receipt of an important grant from the Rosenau Family Research Foundation (RFRF). This grant, titled "Re: Parent-Reported Disease Burden in Infantile and Late Onset Krabbe Disease; A Comparison of Quality-of-Life
- [Passage Bio Announces Third Gene Therapy Development Program in Krabbe Disease and Supports Million Dreams Fundraising Gala](https://krabbeconnect.org/2019/05/passage-bio-announces-third-gene-therapy-development-program-in-krabbe-disease-and-supports-million-dreams-fundraising-gala/) - PHILADELPHIA, May 30, 2019 (GLOBE NEWSWIRE) — Passage Bio, a genetic medicines company developing AAV-delivered gene therapies for the treatment of rare monogenic central nervous system diseases, today announced that its third clinical trial program will be for infantile Krabbe disease, an inherited disease that causes progressive damage to the nervous system. Link to Full
- [Passage Bio's PBKR03 Receives Orphan Drug and Rare Pediatric Disease Designations from FDA for Treatment of Krabbe Disease](https://krabbeconnect.org/2020/10/passage-bios-pbkr03-receives-orphan-drug-and-rare-pediatric-disease-designations-from-fda-for-treatment-of-krabbe-disease/) - PHILADELPHIA, Oct. 28, 2020 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (NASDAQ: PASG), a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system disorders, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug and Rare Pediatric Disease (RPD) designations to PBKR03 for the treatment of Krabbe disease (Globoid Cell Leukodystrophy).
- [FDA Clears IND Application for Passage Bio's Gene Therapy Candidate PBKR03 for Treatment of Patients with Infantile Krabbe Disease, A Rare Pediatric Disorder with No Approved Disease-Modifying Treatment Options](https://krabbeconnect.org/2021/02/fda-clears-ind-application-for-passage-bios-gene-therapy-candidate-pbkr03-for-treatment-of-patients-with-early-infantile-krabbe-disease-a-rare-pediatric-disorder-with-no-approved-disease-modifying/) - PHILADELPHIA, Feb. 08, 2021 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (Nasdaq: PASG), a genetic medicines company focused on developing transformative therapies for rare monogenic central nervous system (CNS) disorders, today announced that the U.S. Food and Drug Administration (FDA) has cleared an investigational new drug (IND) application for PBKR03, an adeno-associated virus (AAV)-delivery gene therapy
- [FDA Fast Track, ODD, and RPDD Designations for FBX-101 Gene Therapy for Patients with Krabbe Disease](https://krabbeconnect.org/2021/02/fda-fast-track-odd-and-rpdd-designations-for-fbx-101-gene-therapy-for-patients-with-krabbe-disease/) - COLUMBUS, OHIO, February 16, 2021 - Forge Biologics Inc., a fully integrated clinical stage gene therapy manufacturing and development company, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track, Orphan Drug, and Rare Pediatric Disease (RPD) designations to FBX-101 for the treatment of patients with Krabbe disease. Forge is now actively recruiting patients
- [U.S. Food and Drug Administration Grants Fast Track Designation to Three Passage Bio Gene Therapy Candidates Targeting Rare CNS Disorders](https://krabbeconnect.org/2021/03/u-s-food-and-drug-administration-grants-fast-track-designation-to-three-passage-bio-gene-therapy-candidates-targeting-rare-cns-disorders/) - PHILADELPHIA, March 8, 2021 (GLOBE NEWSWIRE) -- Passage Bio, Inc., (Nasdaq: PASG), a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system (CNS) disorders, today announced the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to the company's three lead investigational gene therapies: PBGM01 for the treatment
- [Passage Bio Receives European Commission Orphan Designation for PBKR03 for Treatment of Krabbe Disease](https://krabbeconnect.org/2021/04/passage-bio-receives-european-commission-orphan-designation-for-pbkr03-for-treatment-of-krabbe-disease/) - PHILADELPHIA, April 5, 2021 (GLOBE NEWSWIRE) -- Passage Bio, Inc., (Nasdaq: PASG), a clinical-stage genetic medicines company focused on developing transformative therapies for rare monogenic central nervous system (CNS) disorders, today announced that the European Commission has granted Orphan designation for PBKR03, an adeno-associated virus (AAV)-delivery gene therapy for the treatment of Krabbe disease (Globoid
- [Passage Bio Announces Closure of Krabbe Disease Program](https://krabbeconnect.org/2022/11/passage-bio-announces-closure-of-krabbe-disease-program/) - Our Krabbe disease community received some disappointing news today. PassageBio will stop further clinical development of PBKR03 for Krabbe disease. "There's no doubt this news is upsetting but we won't allow it to disrupt our momentum in achieving advances in research and patient care for those impacted by Krabbe disease. However, I do yearn for the
- [Forge Biologics Announces Updated Positive Clinical Data in RESKUE, a Novel Phase 1/2 Gene Therapy Trial for Patients with Krabbe Disease](https://krabbeconnect.org/2022/10/forge-biologics-announces-updated-positive-clinical-data-in-reskue-a-novel-phase-1-2-gene-therapy-trial-for-patients-with-krabbe-disease/) - COLUMBUS, OHIO - Forge Biologics, a gene therapy-focused contract development and manufacturing organization, announced today that Chief Medical Officer Maria Escolar, M.D, MS., presented updated clinical data from the RESKUE Phase 1/2 clinical trial for FBX-101-the Company's novel gene therapy for the treatment of patients with Krabbe disease. Link to Full Release
- [Forge Biologics Reports Positive FBX-101 Clinical Updates for Patients with Krabbe Disease at WorldSymposium](https://krabbeconnect.org/2023/02/forge-biologics-reports-positive-fbx-101-clinical-updates-for-patients-with-krabbe-disease-at-worldsymposium/) - COLUMBUS, OHIO - Forge Biologics, a genetic medicines development and manufacturing organization, announced today that Maria Escolar, M.D., Chief Medical Officer, will present updated data from the RESKUE Phase 1/2 clinical trial for FBX-101, the Company's novel AAV gene therapy for the treatment of patients with Krabbe disease, during the 19th Annual WORLDSymposium being held
- [Joni Knapper – Grandparent of Krabbe Disease Hero](https://krabbeconnect.org/2022/12/joni-knapper-grandparent-of-krabbe-disease-hero/) - This is my story about loving and losing my fourth grandchild, Saylor Irene. Saylor was the precious first born of our daughter, Betsy and her husband, Evan. We are blessed to live about five minutes from Betsy, Evan and Saylor. I am a former NICU nurse and have been a pediatric RN for 42 years.
- [Tammy Feldt - Grandparent of Krabbe Disease Hero](https://krabbeconnect.org/2022/12/tammy-feldt-grandparent-of-krabbe-disease-hero/) - My grandson is Dawson Luke Feldt and he was a Krabbe Warrior. I am his paternal grandmother and I was fortunate enough to be a constant in Dawson’s short life. I lived about 45 minutes away. Dawson was the most beautiful baby and was born healthy (or so we thought). He started not reaching milestones
- [Debbie and Steve McIntosh – Grandparent of Krabbe Disease Hero](https://krabbeconnect.org/2023/02/debbie-and-steve-mcintosh-grandparent-of-krabbe-disease-hero/) - July 2019 was filled with excitement...a new grandchild on the way. He arrived on July 16, normal delivery, mom and baby did great. He was named Dawson Luke. We had no idea within 6 months we would be devastated by hearing a foreign word at a called family meeting with parents and grandparents - Krabbe.
- [Nancy and Darrell Cotterill - Grandparent of Krabbe Disease Hero](https://krabbeconnect.org/2023/04/nancy-and-darrell-cotterill-grandparent-of-krabbe-disease-hero/) - When Collin got sick, we spent many overnights and weekends with them as a family. We didn’t have a big part in Collin’s physical care because he always looked more comfortable in his parents’ arms and eventually in the arms of his nurses. We could tell he preferred his parents touch above all else. I
- [Becky Lebow – Grandparent of Krabbe Disease Hero](https://krabbeconnect.org/2023/05/becky-lebow-grandparent-of-krabbe-disease-hero/) - On September 18, 2017, Mila Rosaleigh Lebow was born at Portsmouth (Virginia) Naval Hospital. All of the nurses were so taken with her. They stated unequivocally that Mila was “perfect”. I was happy to share the moments around her birth. Our son*, Wes, is in the Navy and missed the birth of Mila’s older brother,
- [Ken and DeAnne Close – Grandparent of Krabbe Disease Hero](https://krabbeconnect.org/2023/05/ken-and-deanne-close-grandparent-of-krabbe-disease-hero/) - We are Ken and DeAnne Close, parents of Lesa Brackbill (husband Brennan) and grandparents of Tori. When we found out that we were going to be grandparents we were overjoyed. Because of our own special experiences with our grandparents, finally becoming grandparents ourselves was something we had waited for and looked forward to. Our daughter,
- [Diana Moore – Grandparent of Krabbe Disease Hero](https://krabbeconnect.org/2023/08/diana-moore-grandparent-of-krabbe-disease-hero/) - My name is Diana Moore and I live in Springfield Missouri with my son Ferrell and my granddaughter Regann. At three days old, Ferell received a call from the doctor at Children’s hospital saying Regann had Krabbe Disease which was identified through newborn screening. The doctor said it was critical we come to the hospital
- [Audrey Cushman  – Grandparent of Krabbe Disease Hero](https://krabbeconnect.org/2023/05/audrey-cushman-grandparent-of-krabbe-disease-hero/) - This is my story of my beautiful grandbaby, Collin. My grandson and his parents (my son and daughter-in-law) live over 100 miles away, so it was a good 2 hour drive. They were here for a visit in October after Collin started to show signs something was wrong. He cried most of the time and
- [Mary Meisner – Grandparent of Krabbe Disease Hero](https://krabbeconnect.org/2023/08/mary-meisner-grandparent-of-krabbe-disease-hero/) - On March 19, 2002 our 2nd grandchild Belle Menne was born perfect in every way! For 12 months she developed her fun personality and just when she was about to take her first steps, a subtle shift began to emerge. She began to regress, and within a short period of time she was diagnosed with
- [Long-term Improvements in Lifespan and Pathology in CNS and PNS After BMT Plus One Intravenous Injection of AAVrh10-GALC in Twitcher Mice.](https://krabbeconnect.org/2018/05/long-term-improvements-in-lifespan-and-pathology-in-cns-and-pns-after-bmt-plus-one-intravenous-injection-of-aavrh10-galc-in-twitcher-mice/) - Rafi MA, et al. Long-term Improvements in Lifespan and Pathology in CNS and PNS After BMT Plus One Intravenous Injection of AAVrh10 GALC in Twitcher Mice. Molecular Therapy. Volume 23, Issue 11, November 2015, Pages 1681-1690. Click here to view publication Summary Hematopoietic stem cell transplant (HSCT) is the standard of care for patients with presymptomatic infantile-onset Krabbe
- [Psychosine, a marker of Krabbe phenotype and treatment effect.](https://krabbeconnect.org/2017/07/psychosine-a-marker-of-krabbe-phenotype-and-treatment-effect/) - Escolar ML, Kiely BT, Shawgo E, et al. Psychosine, a marker of Krabbe phenotype and treatment effect. Mol Genet Metab. 2017;121(3):271-278. Click here to view publication Background Newborn screening (NBS) for Krabbe disease (KD) has recently started in a number of US states. Much of the controversy surrounding NBS for KD has centered on the
- [A prospective natural history study of Krabbe disease in a patient cohort with onset between 6 months and 3 years of life](https://krabbeconnect.org/2019/03/a-prospective-natural-history-study-of-krabbe-disease-in-a-patient-cohort-with-onset-between-6-months-and-3-years-of-life/) - Nicholas Bascou, Anthony DeRenzo, Michele D. Poe and Maria L. Escolar Bascouet al. Orphanet Journal of Rare Diseases (2018) 13:126 Click here to view publication Background Natural history studies are an essential part to understanding rare genetic disorders and they play a very important role in evaluating the efficacy of newly developed treatments such as
- [Intrathecal Administration of AAV/GALC Vectors in 10-11 Day old Twitcher Mice Improves Survival and is Enhanced by Bone Marrow Transplant.](https://krabbeconnect.org/2018/05/intrathecal-administration-of-aav-galc-vectors-in-10-11-day-old-twitcher-mice-improves-survival-and-is-enhanced-by-bone-marrow-transplant/) - Download a PDF of this Article Karumuthil-Melethil S, Marshall MS, Heindel C, Jakubauskas B, Bongarzone E, Gray SJ. Intrathecal Administration of AAV/GALC Vectors in 10-11 Day old Twitcher Mic Improves Survival and is Enhanced by Bone Marrow Transplant. Journal of neuroscience research. 2016;94(11):1138-1151. doi:10.1002/jnr.23882. Click here to view publication Background Hematopoietic stem cell transplant (HSCT) is the standard
- [Early progression of Krabbe disease in patients with symptom onset between 0 and 5 months](https://krabbeconnect.org/2019/02/early-progression-krabbe-disease-patients-study/) - Maria L. Beltran-Quintero, Nicholas A. Bascou, Michele D. Poe, David A. Wenger, Carlos A. Saavedra-Matiz, Matthew J. Nichols and Maria L. Escolar Orphanet Journal of Rare Diseases 2019 14:46 Click here to view publication Background Krabbe disease is a rare neurological disorder caused by a deficiency in the lysosomal enzyme, β-galactocerebrosidase, resulting in demyelination of the central and peripheral nervous systems. If left without treatment, Krabbe disease results in
- [Developmental Outcomes of Cord Blood Transplantation for Krabbe Disease](https://krabbeconnect.org/2018/08/matthew-d-wright-et-al/) - Matthew D. Wright, Michele D. Poe, Anthony DeRenzo, Shilpa Haldal, Maria L. Escolar. Developmental outcomes of cord blood transplantation for Krabbe disease; a 15-year study Neurology Aug 2017, 10.1212/WNL.0000000000004418; DOI:10.1212/WNL.0000000000004418. Click here to view publication Background Hematopoietic stem cell transplantation (HSCT) is the standard of care for infantile-onset Krabbe disease and has been shown to
- [Screening for Krabbe disease: The first 2 years' experience](https://krabbeconnect.org/2019/10/screening-for-krabbe-disease-the-first-2-years-experience/) - Pannuzzo, G, Graziano, ACE, Avola, R, Drago, F, Cardile, V. Screening for Krabbe disease: The first 2 years' experience.Acta Neurol Scand . 2019; 00: 1– 7. Click here to view publication Background Globoid cell leukodystrophy or Krabbe disease (KD) is a lysosomal storage disorder characterized by a deficiency in galactosylceramidase (GALC) which breaks down (hydrolyses)
- [The critical role of psychosine in screening, diagnosis, and monitoring of Krabbe disease.](https://krabbeconnect.org/2020/02/the-critical-role-of-psychosine-in-screening-diagnosis-and-monitoring-of-krabbe-disease/) - Guenzel AJ, Turgeon CT, Nickander KK, White AL, Peck DS, Pino GB, Studinski AL, Prasad VK, Kurtzberg J, Escolar ML, Lasio MLD, Pellegrino JE, Sakonju A, Hickey RE, Shallow NM, Ream MA, Orsini JJ, Gelb MH, Raymond K, Gavrilov DK, Oglesbee D, Rinaldo P, Tortorelli S, Matern D Genetics in medicine : official journal of
- [Reduced-intensity single-unit unrelated cord blood transplant with optional immune boost for nonmalignant disorders.](https://krabbeconnect.org/2020/07/reduced-intensity-single-unit-unrelated-cord-blood-transplant-with-optional-immune-boost-for-nonmalignant-disorders/) - Vander Lugt et al, Reduced-intensity single-unit unrelated cord blood transplant with optional immune boost for nonmalignant disorders. Blood Advances 2020; 4 (13): 3041-3052 Click here to view publication Background Historically, treatment-related morbidity and mortality rates have limited referrals for patients who may benefit from allogeneic hematopoietic stem cell transplantation (HSCT). A novel chemotherapy-based reduced-intensive conditioning
- [Krabbe disease successfully treated via monotherapy of intrathecal gene therapy](https://krabbeconnect.org/2020/08/krabbe-disease-successfully-treated-via-monotherapy-of-intrathecal-gene-therapy/) - Bradbury AM, Bagel JH, Nyugen D,et al. Krabbe disease successfully treated via monotherapy of intrathecal gene therapy. J Clin Invest. 2020. Click here to view publication Background Although effective therapies for central nervous system (CNS) disorders, like infantile-onset Krabbe disease (GLD) are being developed, the current inability to treat peripheral neuropathies continues to impact the
- [Patient Pathways](https://krabbeconnect.org/2020/10/patient-journey/)
- [Patient Resources](https://krabbeconnect.org/2020/10/patient-resources/)
- [Together (KrabbeConnect)](https://krabbeconnect.org/2020/10/together-krabbeconnect/) - "Together" is a song written by board member, Dawn Laney, and performed by Marc Gunn. It originally aired on October 10, 2020 during the "A Million Dreams Gala."
- [#curekrabbe "A Million Dreams" Gala](https://krabbeconnect.org/2020/10/curekrabbe-a-million-dreams-gala/) - Please join us live on October 10th, 2020 for our, "A Million Dreams," gala as KrabbeConnect highlights patient stories, embraces the generosity of our corporate sponsors, pays tribute to those patients diagnosed with Krabbe disease, and fundraises to the max to reach a day in which each patient can live a life free of Krabbe
- [Nikola's Krabbe Disease Story: Extended Interview](https://krabbeconnect.org/2020/10/nikolas-krabbe-disease-story-extended-interview/) - Dragon and Lana, parents of Nikola, sit down with KrabbeConnect, to share their Krabbe disease journey. Krabbe disease is a rare neurological disease that results from the loss of the protective covering (myelin sheath) surrounding nerve cells. Dragon and Lana, residents of Virginia, share their story with the hopes of further educating the medical community
- [Transplant Alone is Not a Cure](https://krabbeconnect.org/2020/10/transplant-alone-is-not-a-cure/) - A 3-minute video with Krabbe disease expert, Dr. Joanne Kurtzberg, and 4 amazing transplanted Krabbe disease patients. We're so thankful for transplant to help patients with this rare neurological disease live a better life. Yet, it's important we share this video as transplanted patients need additional therapies to ensure they can continue living their best
- [Long-term neurodevelopmental outcomes of hematopoietic stem cell transplantation for late-infantile Krabbe disease.](https://krabbeconnect.org/2021/04/long-term-neurodevelopmental-outcomes-of-hematopoietic-stem-cell-transplantation-for-late-infantile-krabbe-disease/) - kIsabel C. Yoon, Nicholas A. Bascou, Michele D. Poe, Paul Szabolcs, Maria L. Long-term neurodevelopmental outcomes of hematopoietic stem cell transplantation for late-infantile Krabbe disease.Blood 2021; 137 (13): 1719–1730 Click here to view publication Background Although new therapies are on the horizon for patients with Krabbe disease, currently hematopoietic stem cell transplantation (HSCT) is the
- [Benefits of Newborn Screening and Hematopoietic Cell Transplant in Infantile Krabbe Disease](https://krabbeconnect.org/2022/01/benefits-of-newborn-screening-and-hematopoietic-cell-transplant-in-infantile-krabbe-disease/) - Page KM, Ream MA, Rangarajan HG, et al. Benefits of Newborn Screening and Hematopoietic Cell Transplant in Infantile Krabbe Disease [published online ahead of print, 2022 Jan 18]. Blood Adv. 2022. Read the full article. The following publication highlights the benefits and unique challenges associated with newborn screening (NBS) for early-onset Krabbe disease (also known
- [Improved brain pathology and progressive peripheral neuropathy in a 15 year old survivor of infantile Krabbe disease treated with umbilical cord transplantation](https://krabbeconnect.org/2022/07/improved-brain-pathology-and-progressive-peripheral-neuropathy-in-a-15-year-old-survivor-of-infantile-krabbe-disease-treated-with-umbilical-cord-transplantation/) - Kofler J, Beltran-Quintero ML, Rugari A, et al. Improved Brain Pathology and Progressive Peripheral Neuropathy in a 15-Year-Old Survivor of Infantile Krabbe Disease Treated With Umbilical Cord Transplantation. Front Mol Neurosci. 2022;15:888231. Click here to view the publication Overview Hematopoietic stem cell transplantation (HSCT), most commonly in the form of umbilical cord blood transplantation (UCBT),
- [Krabbe Disease - An Autosomal Recessive Disease](https://krabbeconnect.org/2023/04/krabbe-disease-an-autosomal-recessive-disease/) - How did this happen? How did my child inherit Krabbe disease? You’re not alone in asking this question. This is one of the most common questions asked when one learns that their child or loved one has been diagnosed with Krabbe disease. To learn more about how an individual inherits Krabbe disease, follow the link

## Pages

- [Home](https://krabbeconnect.org/) - KrabbeConnect’s goal is to achieve Krabbe disease eradication through cooperation between patients, advocacy groups, clinicians, researchers and government.
- [Krabbe Disease Types](https://krabbeconnect.org/krabbe-disease/newly-diagnosed/krabbe-disease-types/) - Krabbe disease is classified in four types: Infantile Krabbe Disease, Late Onset Krabbe Disease, Juvenile Onset Krabbe Disease and Adult Onset Krabbe Disease.
- [Donate to KrabbeConnect, Programs and Events](https://krabbeconnect.org/donate/donate-programs-events/) - KrabbeConnect offers a variety of opportunities for donations from special events, donor advised funds and #curekrabbe programs to support our vision and mission statements. Help us in making a difference in the lives of those affected by Krabbe disease by making a donation today! General Donations for KrabbeConnect, Events and Other Programs KrabbeConnect exists entirely
- [Donate to a Family Advised Fund](https://krabbeconnect.org/donate/donate-to-a-family-advised-fund/) - KrabbeConnect offers families the opportunity to establish a Family Advised Fund - a specific fund set up under in the honor/memory of a family member affected by KrabbeConnect to strengthen groundbreaking research, contribute towards a special project, or simply to aid other impactful efforts in the Krabbe disease space. KrabbeConnect presents projects that need help
- [Do Not Sell My Personal Information](https://krabbeconnect.org/do-not-track/) - Please fill out the form below if you wish to remove yourself from our records.
- [Cookie Policy](https://krabbeconnect.org/cookie-policy/) - This page provides comprehensive information about how we use cookies on our website to enhance your browsing experience, improve website performance, and deliver personalized content. Cookies are small text files that are stored on your device when you visit our site. They help us understand how visitors interact with our website, allowing us to offer
- [Experts in Research](https://krabbeconnect.org/research-studies-trials/experts-krabbe-research/) - The individuals featured on this page have devoted a significant portion of their careers to advancing the understanding, diagnosis, treatment, and care of Krabbe disease. Through decades of scientific research, clinical care, newborn screening advancements, and therapy development, these experts have helped shape the progress being made for patients and families impacted by this rare
- [Robert Thompson Stone, MD, FAAN](https://krabbeconnect.org/research-studies-trials/experts-krabbe-research/robert-thompson-stone-md-faan/) - Associate Professor of Neurology and PediatricsUniversity of Rochester What first drew you to Krabbe disease—and why have you stayed? I was initially drawn to Krabbe disease through my work in newborn screening. In New York, Krabbe has been part of newborn screening since 2006, but for a long time we didn’t fully understand how to
- [Steven Gray, Ph.D.](https://krabbeconnect.org/research-studies-trials/experts-krabbe-research/steven-gray-ph-d/) - Professor of Pediatrics and Gene Therapy Program DirectorUT Southwestern Medical Center What first drew you to Krabbe disease—and why have you stayed? I got pulled into this field by colleagues who thought the gene therapy technology I was working on could be helpful. I've stayed in it because the gene therapy approaches, we've developed look
- [Deepa Rajan, MD](https://krabbeconnect.org/research-studies-trials/experts-krabbe-research/deepa-rajan-md/) - Associate Professor of Pediatrics, University of PittsburghDirector, Children's Center for NeuroGenomics (CCNG)UPMC Children's Hospital of Pittsburgh What keeps you going on the hard days when progress feels slow? The children that I care for. Even in the face of incredibly difficult circumstances, their resilience is remarkable - it is that strength that stays with me
- [Anthony Filiano, Ph.D.](https://krabbeconnect.org/research-studies-trials/experts-krabbe-research/anthony-filiano-ph-d/) - Associated ProfessorDuke University What first drew you to Krabbe disease–and why have you stayed? Krabbe disease exposes a fundamental gap in medicine: we can replace the blood and immune system with transplants, yet we still cannot repair the brain. My lab studies the borders of the brain and how we can open the door to
- [Joanne Kurtzberg, MD](https://krabbeconnect.org/research-studies-trials/experts-krabbe-research/joanne-kurtzberg-md/) - Jerome Harris Distinguished Professor of PediatricsProfessor of PathologyDirector, Marcus Center for Cellular CuresDirector, Carolinas Cord Blood BankCo-Director, Stem Cell Transplant LaboratoryPresident, Cord Blood Association What is one thing you wish every family understood about the work happening right now? Two things: (1) While progress is being made, it’s moving more slowly than we would like.
- [Joseph Orsini, Ph.D.](https://krabbeconnect.org/research-studies-trials/experts-krabbe-research/joseph-orsini-ph-d/) - Research ScientistNew York State Department of Health Newborn Screening Program What first drew you to Krabbe disease—and why have you stayed? My first day on Job, I was presented with a research paper by Dr. Michael Gelb that provided a method to screen for Krabbe disease and was told the governor wanted us to implement
- [Maria Irene Givogri, Ph.D.](https://krabbeconnect.org/research-studies-trials/experts-krabbe-research/maria-irene-givogri-ph-d/) - Research Associate ProfessorUniversity of Illinois, Chicago What first drew you to Krabbe disease—and why have you stayed? I have always been deeply curious and passionate about cell biology and medical science. I am particularly inspired by the remarkable scientific and technological advances that have made it possible to treat and cure a wide range of
- [Chris Lee, Ph.D.](https://krabbeconnect.org/research-studies-trials/experts-krabbe-research/chris-lee-ph-d/) - Faculty ScientistBiomedical Research Institute of New Jersey What first drew you to Krabbe disease—and why have you stayed? It was the first project assigned to me when I started my first postdoc. At the time, my PI was drawn into it because of the simplistic nature of the disease mechanism (monogenetic disorder) and yet the
- [Connect with a Krabbe Disease Expert](https://krabbeconnect.org/krabbe-disease/patient-and-caregivers/connect-clinicial-krabbe-expert/) - Once a neurologist suspects or confirms a diagnosis of Krabbe disease, it is critical to connect with experienced specialists as quickly as possible. Due to the rarity and complexity of this disease, many medical providers may never encounter another patient with Krabbe disease during their careers. Families deserve access to experts who understand the disease
- [KrabbeCURES](https://krabbeconnect.org/community-engagement/krabbecures/) - Make a Difference! Participate in the KrabbeCURES Engagement Study by clicking the button below. Click to Sign up for KrabbeCURES! Krabbe Community United Research and Engagement Study (KrabbeCURES) KrabbeCURES, a collaborative effort between KrabbeConnect and the National Organization for Rare Disorders (NORD), to study globoid cell leukodystrophy (Krabbe disease). KrabbeCURES supports research on Krabbe disease
- [Become a Monthly Donor: Join the Champions for Change Circle](https://krabbeconnect.org/donate/become-a-monthly-donor-join-the-champions-for-change-circle/) - When Krabbe disease impacts a child or family, the needs do not happen just once. They come month after month — from urgent comfort items and adaptive equipment to travel support, family assistance, and patient-centered research efforts. By becoming a monthly donor to KrabbeConnect, you provide steady, reliable support that helps families feel less alone
- [Donate Landing](https://krabbeconnect.org/donate/) - Donate "We make a living by what we get ... but we make a life by what we give." Winston Churchill Support KrabbeConnect, Events and Other Programs Donate Now Donate to Family Advised Funds Donate Now Become a Monthly Donor Donate Now
- [Presentations/Publications](https://krabbeconnect.org/research-studies-trials/krabbeconnect-research/krabbe-connect-publications-presentations/) - At KrabbeConnect, we work hard to help fill gaps in research. Below, we invite you to review the research projects presented at conferences and other projects resulting in a publication. Publications Caregiver-reported disease burden in Krabbe disease: evaluating outcomes of hematopoietic stem cell transplantation by Nicholas Alexander Bascou, Skyler Jackson, Patti Engel, Anne Melchior, Paul
- [Select & Pay for your 10x10 Squares](https://krabbeconnect.org/donate/donate-programs-events/super-bowl-challenge-10x10-square-select-and-pay/) - Benefits Patient Assistance Programs $25 per Square. One number will be assigned to each column and row. The numbers for the columns and rows will be assigned randomly when the grid is filled. These represent the last number in the score of each team. End of 1st QTR: $50.00 Amazon Gift Card End of 2nd
- [Select & Pay for your 5x10 Squares](https://krabbeconnect.org/donate/donate-programs-events/super-bowl-challenge-5x10-square-select-and-pay/) - Benefits #curekrabbe Programs $35 per Square. One number will be assigned to each column and two numbers to each row. The numbers for the columns and rows will be assigned randomly when the grid is filled. These represent the last number in the score of each team. End of 1st QTR: $60.00 Amazon Gift Card
- [Select & Pay for your 5x5 Squares (Board 1)](https://krabbeconnect.org/donate/donate-programs-events/super-bowl-challenge-5x5-square-select-and-pay/) - Benefits KRABBECURES Advancing Research $50 per Square. Two numbers will be assigned to each column and row. The numbers for the columns and rows will be assigned randomly when the grid is filled. These represent the last number in the score of each team. End of 1st QTR: $75.00 Amazon Gift Card End of 2nd
- [Select & Pay for your 5x5 Squares (Board 2)](https://krabbeconnect.org/donate/donate-programs-events/select-pay-for-your-5x5-squares-board-2/) - Benefits KRABBECURES Advancing Research $50 per Square. Two numbers will be assigned to each column and row. The numbers for the columns and rows will be assigned randomly when the grid is filled. These represent the last number in the score of each team. End of 1st QTR: $75.00 Amazon Gift Card End of 2nd
- [Thanks for Playing!](https://krabbeconnect.org/donate/thanks-for-playing/) - Thank you for your purchase, your square selections have been submitted. You will receive an email from KrabbeConnect.org noting when further information will be available on your grid square purchase. Interested in purchasing additional squares on other grids? Select your grid using the buttons below: 10x10 Squares 5x10 Squares 5x5 Squares (Board 1) 5x5 Squares (Board
- [KrabbeConnect Bylaws & IRS Forms](https://krabbeconnect.org/about-krabbeconnect/bylaws-irsforms/) - Bylaws Please click to find a download PDF version of the form you selected. KrabbeConnect Bylaws KrabbeConnect IRS Forms Please click to find a download PDF version of the form you selected. KrabbeConnect 2024 - IRS FORM 990 KrabbeConnect 2023 - IRS FORM 990 KrabbeConnect 2022 - IRS FORM 990 KrabbeConnect 2021 - IRS FORM
- [Krabbe Disease Newborn Screening](https://krabbeconnect.org/krabbe-disease/krabbe-disease-newborn-screening/) - Krabbe Disease Newborn Screening is crucial for the early diagnosis, intervention and treatment of Krabbe disease.
- [KrabbeConnect Holiday Grant Program](https://krabbeconnect.org/community-engagement/krabbeconnect-patient-assistance-program/apply-holiday-gift-program/) - As the holiday season approaches, we understand that it can be a time of both joy and challenge for families affected by Krabbe disease. We believe in the power of community, support, and sharing love during this special time. We are excited for the 4th year of the "Krabbe Family Holiday Grant Program," an initiative
- [Shop](https://krabbeconnect.org/shop/) - The KrabbeConnect Store is Offline for Maintenance. Please Contact Us directly if you would like to make a purchase.
- [Juvenile Onset Krabbe Disease (JOKD)](https://krabbeconnect.org/krabbe-disease/juvenile-onset-krabbe-disease-jokd/) - Juvenile Onset Krabbe Disease (JOKD) is a less common form of Krabbe disease, with the onset of symptoms appearing between 4 and 18 years of age.
- [Late Onset Krabbe Disease (LIKD)](https://krabbeconnect.org/krabbe-disease/late-onset-krabbe-disease-lokd/) - Late Onset Krabbe Disease (LIKD) is a common form of Krabbe disease, with the onset of symptoms appearing between 13 and 36 months of age.
- [Newborn Blood Test Screening Can Save Lives](https://krabbeconnect.org/newborn-blood-test-screening-can-save-lives/) - The Newborn Blood Test Screening panel is a routine series of tests that can detect serious medical conditions early in life, often before symptoms appear.
- [KrabbeConnect Board Members](https://krabbeconnect.org/about-krabbeconnect/board-members/) - The KrabbeConnect Board of Directors helps bridge the gap between Krabbe disease science and patient knowledge.
- [We Look Forward to Seeing You at the ULF Family Conference!](https://krabbeconnect.org/community-engagement/apply-for-the-krabbeconnect-stipend-to-attend-the-ulf-family-conference/we-look-forward-to-seeing-you-at-the-ulf-family-conference/) - Thanks for submitting your request for a KrabbeConnect stipend. We will be in touch once we review your application (usually within one or two business days). If you have any questions or concerns, or if you do not hear from us after a few days, please contact us at info@krabbeconnect.org. We look forward to seeing
- [Apply for the KrabbeConnect Stipend to Attend the ULF Family Conference](https://krabbeconnect.org/community-engagement/apply-for-the-krabbeconnect-stipend-to-attend-the-ulf-family-conference/) - Please fill out the form below to request a stipend from KrabbeConnect to attend the ULF Family Conference. If you have any questions or issues, please contact us at info@krabbeconnect.org.
- [Education, Resources, YouTube & News](https://krabbeconnect.org/education-resources-news/) - Education, Resources & News Welcome to our education, resources, and news hub dedicated to fostering knowledge, offering invaluable resources, and keeping you updated with the latest developments in the realm of Krabbe disease. In this area, you can tune into a webinar, watch an informational video, or explore the curated collection of news articles and
- [Krabbe Disease Diagnosis & Treatment](https://krabbeconnect.org/krabbe-disease/newly-diagnosed/testing-treatment-and-clinical-care/) - How to Test for Krabbe Disease? A neurologist or nervous system specialist becomes part of the care team once a patient is thought to have a neurological concern. Your doctor may conduct one or more of the test/s listed below to confirm a diagnosis for Krabbe disease: An MRI to look for abnormalities and white
- [Research & Studies](https://krabbeconnect.org/research-studies-trials/) - Research & Studies Every day, with our vision, KrabbeConnect will support the families, caregivers, and most importantly, the patients affected by Krabbe disease, by bridging the gap between science and patient knowledge. According to the National Center for Advancing Translational Sciences (NCATS), research development faces many hurdles; the complexity in diagnosing rare diseases, widely dispersed
- [KrabbeConnect’s Mission & Vision](https://krabbeconnect.org/about-krabbeconnect/mission-vision/) - The mission of KrabbeConnect is to be the source of comprehensive information and access to resources for patients with Krabbe disease.
- [Ella’s Welcome Wish](https://krabbeconnect.org/programs-events/ellas-welcome-wish/) - Supporting Families on Their Krabbe Journey At Ella’s Welcome Wish, we understand how overwhelming it can be for families when they receive the news that their loved-one has been diagnosed with Krabbe disease. This program was organized to offer a warm welcome during one of the most difficult times in a family’s life. At Ella’s
- [Krabbe Disease Clinical Trials](https://krabbeconnect.org/research-studies-trials/krabbe-clinical-trials/) - KrabbeConnect works to arm those impacted by Krabbe disease with valuable information to ensure families have all the information to enhance disease management all in one spot-HERE. Information is power yet can be overwhelming to understand and grasp. For this reason, it is imperative for families and patients considering participation in any clinical trial to
- [Adult Onset Krabbe Disease (AOKD)](https://krabbeconnect.org/krabbe-disease/adult-onset-krabbe-disease-aokd/) - Adult Onset Krabbe Disease (AOKD) is a progressive degenerative disease, with symptoms presenting in adults over the age of 19.
- [Infantile Krabbe Disease (IKD)](https://krabbeconnect.org/krabbe-disease/early-infantile-krabbe-disease-eikd/) - Infantile Krabbe Disease (IKD) is the most severe form of Krabbe disease, with the onset of symptoms appearing between birth and 13 months of age.
- [Krabbe Disease Patient Support Team](https://krabbeconnect.org/krabbe-disease/patient-and-caregivers/krabbe-disease-patient-support-team/) - Navigating a rare disease is often challenging and overwhelming. Whether you are a parent, sibling, grandparent, spouse, the individual directly living with Krabbe disease, or you fulfill another special role, each person often experiences feelings of isolation, stress, and anxiety for themselves or for the special person living with ongoing health challenges. Whatever your role
- [KrabbeConnect Library](https://krabbeconnect.org/education/krabbeconnect-library/) - KrabbeConnect Library A resource library to inform patients, caregivers, and others, on the research conducted on Krabbe Disease. A Krabbe disease resource library at your fingertips! Scientific research enhances diagnostic tools and advances treatments yet, published articles often result in access and medical dialect barriers. The library, currently in the initial stages, will alleviate this
- [Krabbe Disease Publications Library](https://krabbeconnect.org/education/krabbe-disease-publications-library/) - The KrabbeConnect Disease Publication Library was established to inform patients, caregivers, and others, on the research conducted on Krabbe disease.
- [Experts in Krabbe Disease Newborn Screening](https://krabbeconnect.org/krabbe-disease/experts-in-krabbe-disease-newborn-screening/) - These individuals are experts in the field of Krabbe disease newborn screening. Their scientific discoveries and related studies continue to improve diagnosis.
- [Krabbe Disease Newborn Screening State-by-State Fact Sheets](https://krabbeconnect.org/krabbe-disease/krabbe-disease-newborn-screening/krabbe-disease-newborn-screening-state-fact-sheets/) - Contact us to see how you can help support the efforts in getting Krabbe added to your state's newborn screening panel: Info@krabbeconnect.org.
- [Krabbe Disease Scientific Advisory Council](https://krabbeconnect.org/about-krabbeconnect/krabbeconnect-scientific-advisory-council/) - KrabbeConnect’s Scientific Advisory Council is made up of experts in the fields of medicine, genetics, and Krabbe disease research.
- [About KrabbeConnect](https://krabbeconnect.org/about-krabbeconnect/) - KrabbeConnect works to advance krabbe disease research, provide family support, enhance education, and raise awareness — one step at a time.
- [KrabbeConnect Partners](https://krabbeconnect.org/about-krabbeconnect/partners/) - KrabbeConnect has partnered with organizations and people in the rare disease space for the purpose of advancing our mission and meeting our goals.
- [Contact KrabbeConnect](https://krabbeconnect.org/about-krabbeconnect/contact-us/) - KrabbeConnect is a group of passionate individuals who want to hear from you!
- [Krabbe Disease Hotline](https://krabbeconnect.org/education/krabbe-disease-hotline/) - Call the KrabbeConnect Disease Hotline at 1 (800) 800-5509 Were you or a loved one recently diagnosed with Krabbe disease? Please call our toll-free number 1 (800) 800-5509 for resources, support, and Krabbe disease experts. Looking for information about our programs and services, please give us a call as well — we're here to help.
- [IKD/LIKD NBS Study](https://krabbeconnect.org/research-studies-trials/nbs-study/) - Parent-Reported Disease Burden in Infantile and Late Onset Krabbe Disease; A Comparison of Quality-of-Life Outcomes in Transplant and Non-or-Late Transplant Patients The study intends to gather valuable insights through parent-reported data, enabling a comprehensive assessment of the challenges faced by individuals living with Krabbe disease. By comparing the quality of life among patients who have
- [Current & Ongoing Events](https://krabbeconnect.org/community-engagement/current-events/) - Current & Ongoing Events
- [Science Spotlight](https://krabbeconnect.org/research-studies-trials/science-spotlight/) - Forge Biologics adds Duke University School of Medicine as a Clinical Trial Site for Krabbe disease
- [KrabbeConnect Patient Assistance Program](https://krabbeconnect.org/community-engagement/krabbeconnect-patient-assistance-program/) - Patients and families living with and/or caring for someone with Krabbe disease face enormous challenges. Out-of-pocket medical expenses, large deductibles, unpaid time away from work, supportive therapeutic interventions not covered by insurance, medical equipment, and monthly over-the-counter supplies are just a few of the many struggles that Krabbe families face daily. Through KrabbeConnect's Patient Assistance
- [Thanks for Joining Our March Madness Pool!](https://krabbeconnect.org/donate/thanks-for-joining-our-march-madness-pool/) - Thank you for your purchase, your payment has been submitted. Check your email for information on how to login / create an account with ESPN and make your bracket choices. Good luck!
- [Tipos de la enfermedad de Krabbe](https://krabbeconnect.org/en-espanol/tipos-de-la-enfermedad-de-krabbe/) - La Enfermedad de Krabbe Infantil Inicio de los síntomas: 0 – 13 meses La enfermedad de Krabbe infantil Clásica es la forma más grave y afecta aproximadamente del 85 al 90% de todos los casos.1 Como ocurre normalmente con muchos trastornos por almacenamiento lisosomal, los bebés pueden parecer normales durante los primeros meses de vida y
- [Donar a programas y eventos](https://krabbeconnect.org/en-espanol/donar-a-programas-y-eventos/) - KrabbeConnect ofrece una variedad de oportunidades para donaciones de eventos especiales, fondos asesorados por donantes y programas #curekrabbe para respaldar nuestra visión y misión. ¡Ayúdenos a marcar una diferencia en las vidas de los afectados por la enfermedad de Krabbe haciendo una donación hoy! Done Ahora, Por Favor KrabbeConnect existe enteramente como una organización de
- [En Español](https://krabbeconnect.org/en-espanol/) - Esperamos que obtenga información valiosa de nuestro sitio web en español KrabbeConnect para ayudarlo a navegar la enfermedad. Debido al costo de los servicios de traducción, este sitio web proporciona información pertinente sobre la enfermedad de Krabbe; sin embargo, no es una réplica completa de nuestro sitio en inglés. Si tiene alguna pregunta o necesita
- [KrabbeConnect YouTube Videos](https://krabbeconnect.org/education/krabbeconnect-videos/) - Want to know more about KrabbeConnect and what we are all about? Take a look at our YouTube Channel to view Educational Videos & Webinars, Events, Tributes, and Patient’s Stories affected by Krabbe disease. You can also view our videos by specific topics: Patient Stories Educational Videos Event Videos Webinars Our YouTube Channel
- [Krabbe Disease Heroes](https://krabbeconnect.org/our-heroes/) - The KrabbeConnect Hero page was created for individuals living and no longer living with Krabbe disease. This page helps others to understand the impact of the disease, helps educate healthcare professionals and companies on the numerous lives affected by Krabbe disease as well as helps provide a place of remembrance. At KrabbeConnect we never want
- [Héroes de la enfermedad de Krabbe](https://krabbeconnect.org/en-espanol/heroes-de-la-enfermedad-de-krabbe/) - La página Héroes de KrabbeConnect fue creada para personas que viven con la enferemdad de Krabbe o ya fallecieron debido a esta enfermedad. Esta página ayuda a otras personas a entender el impacto de la enfermedad de Krabbe, ayuda también a educar a los profesionales de la salud y a la industria farmaceútica acerca de
- [Reconozca a sus héroes](https://krabbeconnect.org/en-espanol/heroes-de-la-enfermedad-de-krabbe/reconozca-a-sus-heroes/) - Para enviarnos una foto de su héroe, proporcione: Una imagen de alta resolución, si es posible El nombre de su héroe y la sigla del primer apellido La ciudad, el estado y el pais del personaje en la foto. Complete el formulario de ‘Autorización para uso de fotografías’ a continuación
- [Trasplante para el tratamiento de la enfermedad de Krabbe](https://krabbeconnect.org/en-espanol/trasplante-para-el-tratamiento-de-la-enfermedad-de-krabbe/) - El trasplante de células madre hematopoyéticas (TCMH) es el trasplante de células madre hematopoyéticas multipotentes, generalmente derivadas de la médula ósea, la sangre periférica o la sangre del cordón umbilical. Dependiendo de dónde se originen las células madre del trasplante, se puede denominar uno de los siguientes: trasplante de médula ósea, sangre del cordón umbilical o trasplante periférico. El
- [KrabbeCURES](https://krabbeconnect.org/en-espanol/krabbecures/) - Marca la diferencia Participe en el estudio de compromiso KrabbeCURES haciendo clic en el botón de abajo. ÚNASE A LA INICIATIVA EN KRABBECURES.IAMRARE.ORG Estudio de participación e investigación de Krabbe Community United (KrabbeCURES) KrabbeCURES, un esfuerzo de colaboración entre KrabbeConnect y la Organización Nacional de Enfermedades Raras (NORD), para estudiar la leucodistrofia de células globoides (enfermedad de
- [Las Pruebas, El Tratamiento y La Atención Clínica](https://krabbeconnect.org/en-espanol/las-pruebas-el-tratamiento-y-la-atencion-clinica/) - ¿Cómo realizar ua prueba diagnóstica de la enfermedad de Krabbe? Un neurólogo o un especialista en el sistema nervioso se convierte en parte del equipo de atención una vez que se considere que el paciente tiene un problema neurológico. Su médico puede realizar una o más de las pruebas que se enumeran a continuación para confirmar un
- [Conéctese con un Experto Clínico](https://krabbeconnect.org/en-espanol/conectese-con-un-experto-clinico/) - Una vez que su neurólogo sospeche o confirme el diagnóstico de la enfermedad de Krabbe es importante que se comuníque con los expertos sobre el tema. Debido a la complejidad de esta rara enfermedad, es probable que su equipo de atención médica rutinaria nuncahaya encontrado a otro paciente con esta condición. Es imperativo que su familiar
- [Solicitud Para Recibir Intercambio de Equipos KrabbeConnect](https://krabbeconnect.org/en-espanol/programa-de-asistencia-al-paciente-krabbeconnect/solicitud-para-recibir-intercambio-de-equipos-krabbeconnect/) - Este programa facilitará el intercambio de equipo médico duradero y suministros no utilizados como soportes, andadores especiales, bombas de alimentación, dispositivos de posicionamiento, fórmula, pañales, oxímetros, etc. Si tiene artículos disponibles para donar,por favor siga estos pasos antes de completando el formulario del programa de intercambio de equipos: Envíe un correo electrónico a info@krabbeconnect.org para informarnos de los artículos
- [Solicitud para Donar al Intercambio de Equipos KrabbeConnect](https://krabbeconnect.org/en-espanol/programa-de-asistencia-al-paciente-krabbeconnect/solicitud-para-donar-al-intercambio-de-equipos-krabbeconnect/) - Este programa facilitará el intercambio de equipo médico duradero y suministros no utilizados como soportes, andadores especiales, bombas de alimentación, dispositivos de posicionamiento, fórmula, pañales, oxímetros, etc. Si tiene artículos disponibles para donar,por favor siga estos pasos antes de completando el formulario del programa de intercambio de equipos: Envíe un correo electrónico a info@krabbeconnect.org para informarnos de los artículos
- [Programa de Suministros Médicos](https://krabbeconnect.org/en-espanol/programa-de-asistencia-al-paciente-krabbeconnect/programa-de-suministros-medicos/) - Este programa se encuentra orientado a proporcionar artículos de necesidad médica a los pacientes que viven con la enfermedad de Krabbe. Para nosotros es importante que los pacientes que padecen esta enfermedad vivan su mejor vida. Algunos ejemplos de elementos cubiertos por este programa son tapetes de enfriamiento, dispositivos de posicionamiento, mantas pesadas, etc. Le
- [Programa de KrabbeConnect de Subvenciones Para los Días Feriados](https://krabbeconnect.org/en-espanol/programa-de-asistencia-al-paciente-krabbeconnect/programa-de-krabbeconnect-de-subvenciones-para-los-dias-feriados/) - A medida que se acercan las fiestas navideñas, sabemos que pueden significar una temporada de alegría como también de grandes retos para las familias afectadas por la enfermedad de Krabbe. Creemos en el poder de la comunidad, del apoyo y del amor compartido en estos momentos tan especiales. Estamos entusiasmados con el segundo año del
- [Programa de KrabbeConnect Para La Asistencia Debido a Dificultades Económicas](https://krabbeconnect.org/en-espanol/programa-de-asistencia-al-paciente-krabbeconnect/programa-de-krabbeconnect-para-la-asistencia-debido-a-dificultades-economicas/) - Este programa ha sido diseñado con la finalidad de proporcionar alivio económico a los pacientes con la enfermedad de Krabbe, o a los cuidadores/tutores con un ser amado que padezca la enfermedad de Krabbe y/o esté sufriendo una adversidad económica o viviendo una larga estadía en el hospital. KrabbeConnect se compromete a ayudar a aliviar
- [Programa de asistencia al paciente KrabbeConnect](https://krabbeconnect.org/en-espanol/programa-de-asistencia-al-paciente-krabbeconnect/) - Programa de asistencia al paciente ¡Una pequeña ayuda cuando más la necesitas! Los pacientes y las familias que viven con la enfermedad de Krabbe o cuidan de alguien que la padece se enfrentan a enormes retos. Los gastos médicos de bolsillo, las grandes franquicias, el tiempo no remunerado de ausencia del trabajo, las intervenciones terapéuticas
- [Línea directa de enfermedad de Krabbe](https://krabbeconnect.org/en-espanol/linea-directa-de-enfermedad-de-krabbe/) - 1 (800) 800-5509 ¿Le han diagnosticado recientemente la enfermedad de Krabbe a usted o a un ser querido? Llame a nuestro número gratuito 1 (800) 800-5509 para obtener recursos, apoyo y expertos en la enfermedad de Krabbe. Si busca información sobre nuestros programas y servicios, llámenos también: estamos aquí para ayudarle.
- [Entendiendo la enfermedad de Krabbe](https://krabbeconnect.org/en-espanol/entendiendo-la-enfermedad-de-krabbe/) - ¿Qué es la enfermedad de Krabbe? La enfermedad de Krabbe (pronunciada krab-é) es un trastorno genético poco común, también conocido como la leucodistrofia de células globoides. La enfermedad de Krabbe se describe como una enfermedad neurológica grave que resulta de la pérdida de la cubierta protectora (vaina de mielina) que rodea las células nerviosas. Esta vaina protectora
- [Conocimientos del Consejero en Genética / Equipo de Atención Médico](https://krabbeconnect.org/en-espanol/conocimientos-del-consejero-en-genetica-equipo-de-atencion-medico/) - ¿Qué es un Consejero en Genética? Un consejero en genética (GC, por sus siglas en inglés) es un proveedor de atención médica que se especializa en ayudar y capacitar a las familias sobre cómo navegar las complejidades de una condición genética. Los consejeros en genética (GC, por sus siglas en inglés) reciben capacitación especializada en
- [Ensayos Clínicos Sobre la enfermedad de Krabbe](https://krabbeconnect.org/en-espanol/ensayos-clinicos-sobre-la-enfermedad-de-krabbe/) - KrabbeConnect trabaja para armar a los afectados por esta enfermedad con valiosa información que garantice a las familias tener acceso a una más completa información a fin de mejorar el manejo de la enfermedad, todo en un mismo lugar: AQUÍ. La información es poder; sin embargo puede ser abrumador y complejo comprender y captar, en
- [Event Videos](https://krabbeconnect.org/education/krabbeconnect-videos/videos-events/)
- [Patient Stories Videos](https://krabbeconnect.org/education/krabbeconnect-videos/videos-patient-stories/)
- [Educational Videos & Webinars](https://krabbeconnect.org/education/krabbeconnect-videos/videos-education/)
- [Programs & Events](https://krabbeconnect.org/programs-events/) - Programs & Events Welcome to our Krabbe disease programs and events hub, dedicated to raising awareness, fostering community, and driving progress in the fight to #curekrabbe. We invite you to explore the different sections highlighting our impactful programs and upcoming events. Each section offers a comprehensive look into our initiatives, support networks, and opportunities for
- [Krabbe Disease](https://krabbeconnect.org/krabbe-disease/) - Krabbe Disease Hello and welcome! In this section we work to provide knowledge and insights to empower those impacted by Krabbe disease. This section is divided into 3 parts: Newly Diagnosed, Patients and Caregivers, and Newborn Screening. Whether you're navigating a recent diagnosis, walking alongside a loved one as a caregiver, or looking to gain
- [Past Events](https://krabbeconnect.org/community-engagement/past-events/) - Past Events Participate in an event and help support Krabbe research and patient support!
- [Listen and Learn Webinars](https://krabbeconnect.org/education/listen-and-learn-webinars/) - KrabbeConnect will host special webinars to keep patients, caregivers, industry stakeholders, non-profit partners, the medical community, and others abreast of important information including but not limited to disease insights, COVID-19 concerns/pre-cautions, new therapies, clinical trial presentations, coping skills, and more! If you have a topic of interest, we want to hear from you. Email us
- [KrabbeConnect YouTube Channel](https://krabbeconnect.org/education/krabbeconnect-youtube-channel/) - Want to know more about KrabbeConnect and what we are all about? Take a look at our YouTube Channel to view informative Webinars, Events, Tributes, and Patient’s Stories affected by Krabbe disease. Here’s a list of just a few things you can watch: Two Sister’s Journey with Krabbe disease: The Moore Family Story Cloud
- [#BeRare Essay](https://krabbeconnect.org/community-engagement/berare-essay/) - Thank you so much for your interest in participating in one of our 2021 Rare Disease Day activities. We are glad you are here! We are inviting those impacted by a leukodystrophy to participate in the #BeRare essay. Krabbe disease is one of more than 50-identified leukodystrophies recognized by the National Institute of Health. Please
- [Glossary](https://krabbeconnect.org/glossary/)
- [Grandparent Stories of Krabbe Heroes](https://krabbeconnect.org/community-engagement/krabbe-grandparents/) - Personal Stories from the grandparents of Krabbe Heroes Navigating Krabbe disease is a family matter. The complexity of care for a loved one diagnosed with a rare disease can also affect grandparents. Grandparents find themselves struggling to navigate how to best support their grandchild and their adult child. They are also sorting out feelings of
- [Patients & Caregivers](https://krabbeconnect.org/krabbe-disease/patient-and-caregivers/) - Patients & Caregivers Being diagnosed with a rare disease is terrifying and difficult, yet, armed with the right information, KrabbeConnect hopes to make the journey slightly less stressful. The information provided here is designed to help patients and caregivers understand what they can expect when a loved one is diagnosed with Krabbe disease. KrabbeConnect is
- [Krabbe Disease Press Releases](https://krabbeconnect.org/research-studies-trials/krabbe-clinical-trials/krabbe-disease-press-releases/)
- [Diríjase a la Caja](https://krabbeconnect.org/revisa/) - [woocommerce_checkout]
- [KrabbeConnect Research](https://krabbeconnect.org/research-studies-trials/krabbeconnect-research/) - Coming soon ...
- [Resources for Clinical Trials](https://krabbeconnect.org/research-studies-trials/krabbe-clinical-trials/resources-for-clinical-trials/) - It's important to be well-informed about your participation in a clinical trial. Use this informational sheet to help ask important questions prior to enrolling in a clinical trial for Krabbe disease. Questions for the Principal Investigator: How many participants have enrolled in this clinical trial thus far? What is the total time commitment to participate
- [Recognize Your Heroes](https://krabbeconnect.org/recognize-your-heroes/) - To submit a photo of your hero, please provide: A high resolution image, if possible The first name you'd like to display and initial of last name The city and state of pictured individual Complete Permission to Use Photograph form below
- [KrabbeConnect Library](https://krabbeconnect.org/krabbeconnect-library/)
- [KrabbeConnect Email Subscription Signup](https://krabbeconnect.org/krabbeconnect-email-subscription-signup/)
- [Krabbe Disease Peer Mentor Story – Kevin Cushman](https://krabbeconnect.org/krabbe-disease/patient-and-caregivers/krabbe-disease-patient-support-team/krabbe-disease-peer-mentor-story-kevin-cushman/) - When my wife was pregnant, I was asked if I wanted a boy or a girl. The answer was always the same, “It doesn’t matter, as long as the baby is healthy.” When Collin came into the world, on December 19, 2010 our doctor said, “he is perfect and healthy.” As his parents, we were
- [Krabbe Disease Peer Mentor Story – Anne Rugari](https://krabbeconnect.org/krabbe-disease/patient-and-caregivers/krabbe-disease-patient-support-team/krabbe-disease-peer-mentor-story-anne-rugari/) - While raising her healthy first child, Philip, Anne Rugari had a second son, Nick, who was diagnosed with Infantile Krabbe Disease at 7 months of age in 1986. Nick was only a year old when he passed away. Anne knew as she was expecting her third child in 1999, that the baby should be tested
- [Resources](https://krabbeconnect.org/krabbe-disease/patient-and-caregivers/resources/) - Resources In keeping with the mission of KrabbeConnect, it’s important we arm you with pertinent information at your fingertips, enabling you to make informed decision that are right for you or your affected loved one. Managing a rare disease is not easy. There are unique challenges and opportunities that require decision on where to obtain
- [Genetic Counselor/Care Team Knowledge](https://krabbeconnect.org/krabbe-disease/patient-and-caregivers/genetic-counselor-care-team-knowledge/) - What Is a Genetic Counselor? A genetic counselor is a healthcare provider who specializes in helping and empowering families on how to navigate the complexities of a genetic condition. Genetic counselor’s (GC) receive specialized training in genetics, communication, counselling, and coordination of services for complex medical conditions like Krabbe disease (KD). Many families living with
- [Advocating for Your Patient](https://krabbeconnect.org/krabbe-disease/patient-and-caregivers/advocating-for-your-patient/) - Patient Care Center: How to Build a Medical Care Team Original Air Date: May 6th, 2020 A 1-hour presentation with guest speaker, Dawn Laney. Dawn juggles many roles and responsibilities at Emory University: Assistant Professor, Genetics Counselor, Director of the Emory Genetic Clinical Trials Center, Program Leader of Emory's Lysosomal Storage Disease Center, and member
- [Understanding Krabbe Disease](https://krabbeconnect.org/krabbe-disease/newly-diagnosed/what-is-krabbe/) - What is Krabbe Disease? Krabbe disease (pronounced krab A), is a rare genetic disorder, also known as globoid cell leukodystrophy. Krabbe disease is described as a severe neurological condition that results from the loss of the protective covering (myelin sheath) surrounding nerve cells. This protective myelin sheath is essential to insulate the nerves and ensure
- [Treatment and Resource Maps](https://krabbeconnect.org/krabbe-disease/newly-diagnosed/patient-journey/) - The Krabbe disease Patient Journey Map works by simplifying the two pathways of Krabbe disease: the symptomatic and the pre-symptomatic journey. The symptomatic pathway relates to patients who receive a diagnosis with the presentation of clinical symptoms such as, severe acid reflux, stiff muscles, seizures, loss of milestones, lower limb weakness, and more. The pre-symptomatic
- [Transplant for Krabbe Disease Therapy](https://krabbeconnect.org/krabbe-disease/newly-diagnosed/transplant-101/) - Hematopoietic Stem Cell Transplantation (HSCT) is the transplantation of multipotent hematopoietic stem cells, usually derived from bone marrow, peripheral blood, or umbilical cord. Depending on where the stem cells originate from the transplantation may be called one of the following: Bone Marrow, Cord Blood, or Peripheral Transplantation. HSCT has been utilized for more than 20
- [Submit Your Krabbe Hero's Story](https://krabbeconnect.org/our-heroes/submit-your-krabbe-heros-story/)
- [Patient Journey Maps and Resources](https://krabbeconnect.org/education/patient-journey-maps-and-resources/) - The Krabbe disease Patient Journey Map works by simplifying the two pathways of Krabbe disease: the symptomatic and the pre-symptomatic journey. The symptomatic pathway relates to patients who receive a diagnosis with the presentation of clinical symptoms such as, severe acid reflux, stiff muscles, seizures, loss of milestones, lower limb weakness, and more. The pre-symptomatic
- [Partner with Us-Family Advised Fund](https://krabbeconnect.org/community-engagement/partner-with-krabbe-connect/) - Each family impacted by Krabbe disease responds differently as we're all uniquely different. As you work to navigate the ups and downs of this life-threatening disease, perhaps you may be feeling empowered to jump into action. You may be asking yourself where can I make the greatest impact? How can I quickly get up-to-speed on
- [Medical Supply Program](https://krabbeconnect.org/community-engagement/krabbeconnect-patient-assistance-program/apply-medical-equipment-program/) - This program works to help provide items of medical necessity to patients living with Krabbe disease. It's important to us that patients living with this disease live their best life. Some examples of items covered under this program are cooling mats, positioning devices, weighted blankets, etc. You have been diagnosed with Krabbe disease or you
- [KrabbeConnect Hardship Assistance Program](https://krabbeconnect.org/community-engagement/krabbeconnect-patient-assistance-program/application-mortgage-rent-assistance/) - This program works to help provide relief to Krabbe disease patients or caregivers/guardians of a Krabbe disease loved one enduring hardship or long hospital stay. KrabbeConnect is committed to helping relieve some anxiety, stress, and financial burden that can come from a medical crisis. If you are a direct caregiver/guardian of a Krabbe disease loved
- [Application to Receive from the KrabbeConnect Equipment Exchange](https://krabbeconnect.org/community-engagement/krabbeconnect-patient-assistance-program/application-receive-equipment-exchange/) - This program will facilitate the exchange of durable medical equipment and unused supplies such as standers, specialty walkers, feeding pumps, positioning devices, formula, diapers, oximeter, etc. If you have items available to donate, please follow these steps before completing the Equipment Exchange Program Form: Send an email to info@krabbeconnect.org to inform us of the items
- [Application to Donate to the KrabbeConnect Equipment Exchange](https://krabbeconnect.org/community-engagement/krabbeconnect-patient-assistance-program/application-donate-equipment-exchange/) - This program will facilitate the exchange of durable medical equipment and unused supplies such as standers, specialty walkers, feeding pumps, positioning devices, formula, diapers, oximeter, etc. If you have items available to donate, please follow these steps before completing the Equipment Exchange Program Form: Send an email to info@krabbeconnect.org to inform us of the items
- [#CureKrabbe Social Media Toolkit](https://krabbeconnect.org/community-engagement/curekrabbe-social-media-toolkit/) - Educate Your Cohort of Followers by Sharing Help us raise awareness about Krabbe disease! We’ve put together a toolkit with images that you can share on your social media channels. Easily download each piece below. Remember to use hashtag #curekrabbe and #krabbedisease2020! Social Media Cover Photos: Profile Picture Frame:
- [Community Engagement](https://krabbeconnect.org/community-engagement/) - Community Engagement KrabbeConnect needs YOU! As a rare disease non-profit foundation, we are honored and excited to work with those who share our passion and mission for Krabbe disease. Working Together to Make a Difference! Together, with your time, talent, and support, we will reach our vision: A day in which each patient receives an early diagnosis,
- [Be The Story - Story Submission Page](https://krabbeconnect.org/be-the-story-story-submission-page/)
- [Terms & Conditions](https://krabbeconnect.org/about-krabbeconnect/terms-conditions/) - These Website Standard Terms and Conditions written on this webpage shall manage your use of our website, KrabbeConnect accessible at krabbeconnect.org. These Terms will be applied fully and affect to your use of this Website. By using this Website, you agreed to accept all terms and conditions written in here. You must not use this
- [Privacy Policy](https://krabbeconnect.org/about-krabbeconnect/privacy-policy/) - KrabbeConnect operates the krabbeconnect.org website, which provides the comprehensive information and access to resources for patients with Krabbe disease (SERVICE). This page is used to inform website visitors regarding our policies with the collection, use, and disclosure of Personal Information if anyone decided to use our Service, the KrabbeConnect website. If you choose to use
- [Education](https://krabbeconnect.org/education/)
- [Newly Diagnosed](https://krabbeconnect.org/krabbe-disease/newly-diagnosed/)

## Events

- [2026 Leukodystrophy & Newborn Screening Awareness Month](https://krabbeconnect.org/kcevent/leukodystrophy-newborn-screening-awareness-month/) - September is Leukodystrophy and Newborn Screening Awareness Month! The goal is to encourage parents, healthcare providers, and policymakers to advocate for the inclusion of more conditions, like leukodystrophy, in routine newborn screening panels.
- [2026 Super Bowl Square Challenge](https://krabbeconnect.org/kcevent/annual-super-bowl-square-football-challenge/) - Super Bowl squares, also known as football squares, are often a fun staple of Super Bowl parties everywhere. In 2018 we initiated this fundraiser as a fun way to drive excitement about our mission at KrabbeConnect which is, to be the source of comprehensive information and access to resources for patients with Krabbe disease. The foundation will drive state-of-the-art research by bridging the gap between science and patient knowledge.
- [2026 Anywhere Strides North Shore Trek to #curekrabbe!](https://krabbeconnect.org/kcevent/anywhere-strides-to-curekrabbe/) - Join Now Anywhere Strides North Shore Trek to #CureKrabbe39 Miles. 10 Days. One Mission. #curekrabbe No matter where you live, you can join the fight against Krabbe disease. During this 10-day challenge, participants work toward completing 39 miles by walking, running, biking, or rolling their wheelchair—at their own pace and in their own community. Inspired
- [2026 Marshall's Miracle Golf Match](https://krabbeconnect.org/kcevent/marshalls-miracle-golf-match/) - Get Tickets Now Saturday, October 3, 2026 The Nine Golf Course9301 SW Bayou Dr.McMinnville, OR 97128 Marshall's Miracle Golf Match was created in honor of Marshall, whose courageous battle with Krabbe disease forever changed his family's life. Marshall's younger brother, Michael, was spared the disease through an extraordinary medical journey made possible by advances in newborn screening
- [2026 Krabbe Family Warrior Meet-up at the United Leukodystrophy Family Conference](https://krabbeconnect.org/kcevent/krabbe-family-warrior-meet-up-at-the-united-leukodystrophy-family-conference/) - This family meet-up will feature Krabbe-specific scientific, advocacy, and family support sessions. This meeting is designed to empower patients and their families by providing information and tools to help navigate Krabbe disease. KrabbeConnect wants you to use this time to connect and learn from others walking in your shoes, hear from the medical and research
- [Tygh’s Strides to #curekrabbe 5K](https://krabbeconnect.org/kcevent/tyghs-strides-to-curekrabbe-5k/) - Lace up, show up, and help us take meaningful strides toward a cure for Krabbe disease! Join us for Tygh’s Strides to #curekrabbe, a family-friendly 5K event bringing our community together to raise awareness, inspire hope, and support families impacted by Krabbe disease. Event Details Date: September 26th, 2026Check-In: 7:30 a.m. - 8:30 a.m. ESTShotgun
- [HYBRID for Hope: Michelle Salvo’s Campaign to #curekrabbe](https://krabbeconnect.org/kcevent/hybrid-for-hope/) - This is more than a race. This is a mission. Michelle and her training partner, Bre, are taking on a HYROX race as part of Hybrid for Hope—turning strength, endurance, and discipline into support for families impacted by Krabbe disease. This cause is deeply personal. Michelle has seen firsthand the strength it takes for families
- [2026 BrAvery for Avery Golf Tournament](https://krabbeconnect.org/kcevent/bravery-for-avery-golf-tournament/) - Come swing a club and make a difference at the 6th Annual BrAvery for Avery Golf Tournament at Valley Country Club in Warwick, RI — a day of golf, community, and hope as we honor Avery’s legacy and help #curekrabbe. Register and Pay This special event honors the life and legacy of Avery Coupe while
- [2026 Putt-Putt to #CureKrabbe](https://krabbeconnect.org/kcevent/putt-putt-to-curekrabbe/) - Join us for a fun evening of miniature golf while giving back to families impacted by Krabbe disease! Putt-Putt to #CureKrabbe brings together community members, professionals, and advocates for friendly competition, connection, and purpose. Purchase Tickets Event Details Date: Friday, March 20th, 2026 Time: 5:30-8:30 PM CST (check-in starts at 4:45) Ticket/Team Cost: $125 per
- [2026 Rare Disease Day](https://krabbeconnect.org/kcevent/rare-disease-day/) - What are you doing for Rare Disease Day 2026? Krabbe disease affects approximately 40 to 50 patients worldwide per year but on Saturday, February 28th we are part of one BIG FAMILY as we celebrate Rare Disease Day along with the 7,000 other rare conditions. Each year, many organizations across the world are involved
- [Be the Story — Krabbe Disease Newborn Screening Campaign](https://krabbeconnect.org/kcevent/be-the-story-krabbe-disease-newborn-screening-campaign/) - Are you interested in being the face of our Krabbe disease newborn screening state fact sheets? We’re on a mission to help bring more education and awareness to Krabbe disease newborn screening. All 50 states must be testing for this condition now to prevent a delayed diagnosis, especially in the most severe forms. You can be the story to help change Krabbe disease. See which states across the USA do not have a Krabbe disease fact sheet. If your state is not listed, we invite you to share your story.
- [2025 Match My Gift to KrabbeConnect](https://krabbeconnect.org/kcevent/match-my-gift-to-krabbeconnect/) - Many families impacted by Krabbe disease often need relief from time to time. Help with a mortgage or rent payment because their loved one has been hospitalized for more than 4 months. Assistance in covering expensive medicines. Financial support for a therapy that brings their loved one pain relief. Or simply just a little holiday boost to feel supported during a burdensome journey navigating Krabbe disease.
- [2025 Krabbe Family Holiday Grant Program](https://krabbeconnect.org/kcevent/krabbe-family-holiday-grant-program/) - As the holiday season approaches, we understand that it can be a time of both joy and challenge for families affected by Krabbe disease. We believe in the power of community, support, and sharing love during this special time. We are excited for the 4th year of the "Krabbe Family Holiday Grant Program," an initiative
- [KrabbeConnect Wine Collection](https://krabbeconnect.org/kcevent/krabbeconnect-wine-collection/) - In October 2023, KrabbeConnect connected with Jennifer Tanner at Old York Cellars Winery to talk about how their group works to give back to philanthropic causes. After a month of discussion and planning, Old York Cellars helped develop a unique collection of wines aimed to raise funds and awareness for Krabbe disease. Shop Online Now
- [See’s Candies to Help #curekrabbe](https://krabbeconnect.org/kcevent/sees-candies-to-help-curekrabbe/) - Sweeten hearts and change lives! Join Krabbeconnect’s Year-Round Candy Drive. Your support will help satisfy sweet cravings and fuel research for those affected by Krabbe disease. This is a great way to send a gift to others while making a difference with your purchase! Participation is simple. Here’s how it works: You can ship
- [Connor’s Kure for Krabbe](https://krabbeconnect.org/kcevent/connors-kure-for-krabbe/) - Purchase Tickets Become a Sponsor Join us for Connor’s Kure for Krabbe, a heartfelt fundraising dinner event held at the stunning Coeur d'Alene Resort overlooking the crystal waters of Lake Coeur d’Alene in Coeur d’Alene, Idaho. This special evening is dedicated to honoring the life of Connor, a brave little boy who courageously fought Krabbe
- [Bloom for a Cure: A Family-Friendly Event at Hershey Gardens](https://krabbeconnect.org/kcevent/bloom-for-a-cure/) - Buy Tickets Online Become a Sponsor Browse Auction Join us for a heartwarming and joyful evening at the beautiful Hershey Gardens as we unite to raise awareness and vital funds for Krabbe disease research. This special event brings families, friends, and community members together in support of a meaningful cause — helping children and families
- [2025 Swing for Saylor Golf Tournament](https://krabbeconnect.org/kcevent/swing-for-saylor-golf-tournament/) - KrabbeConnect is excited to introduce a new and meaningful way to bring our community together—one that raises awareness and vital funds to support families affected by Krabbe disease. We are proud to announce the Inaugural Swing for Saylor Golf Tournament, set to take place on Sunday, June 1st, 2025, at Emerald Greens Golf Course in
- [Ten Candles, Endless Hope: Tori’s Birthday Benefit to #curekrabbe](https://krabbeconnect.org/kcevent/ten-candles-endless-hope/) - This event is new and it’s replacing our “A Million Dreams Gala.” Why? It’s important to KrabbeConnect to move things around the country giving other families the opportunity to witness the generosity of the community and to attend these special events. Welcome to the Ten Candles, Endless Hope event page, dedicated to Tori’s Birthday Benefit in support of the
- [2024 Valentines Day Candy Drive](https://krabbeconnect.org/kcevent/2024-valentines-day-candy-drive/) - Sweeten hearts and change lives! Join Krabbeconnect’s Valentine's Candy Drive. Your support will help satisfies sweet cravings and fuel research for those affected by Krabbe disease. This is a great way to show your love for others while making a difference with your purchase! Participation is simple. Here’s how it works: Visit our KrabbeConnect
- [2024 March Madness](https://krabbeconnect.org/kcevent/march-madness/) - March Madness is Here!! Join our NCAA basketball tournament bracket challenge and have a chance to win some money, have fun, and help support the mission of KrabbeConnect! How to Play Step 1 Purchase Your Brackets Brackets are $10 a piece, and you can buy up to 10 brackets. To get started, just fill out
- [Matching Donation Superhero](https://krabbeconnect.org/kcevent/matching-donation-superhero/) - We need your help! Thanks to an anonymous superhero in our community, every dollar you donate between now and December 31st will be DOUBLED! Your impact just got a mighty boost—help us unlock this generous matching gift and make your contribution go twice as far! Learn how your donation will help KrabbeConnect.
- [#CureKrabbe 30-Day Challenge](https://krabbeconnect.org/kcevent/curekrabbe-30-day-challenge/) - Are you up for a challenge this summer? Our community of patients experience challenges each day due to their Krabbe disease diagnosis. Please join us for our third annual #curekrabbe Challenge: Bridging the Border run, walk, swim, bike event. Help us to help those individuals affected by Krabbe disease. Registration opens April 1st. Challenge runs May 15-June 15th. Register early to to receive your #curekrabbe swag! You can make a difference and help KrabbeConnect get one step closer to #curekrabbe.
- [A Million Dreams Gala](https://krabbeconnect.org/kcevent/a-million-dreams-gala/) - Join us for our 5th annual "A Million Dreams” Gala on Friday, September 8th, 2023! This premium event is fun, educational, and you can party with a purpose. It’s important to celebrate the accomplishments over the past year and fundraise to help fill necessary gaps in research and help families navigate a rare disease. For now, we ask you to simply save this important date. Tickets will be available for purchase after the 4th of July, 2023.
- [Leukodystrophy Awareness Month](https://krabbeconnect.org/kcevent/leukodystrophy-awareness-month/) - September is Leukodystrophy Awareness Month! A leukodystrophy is a type of disease that causes degeneration of the myelin - or "white matter" - of the brain. Every individual requires healthy production of myelin for speed and accuracy of nerve impulses within the brain. Without a healthy myelin sheath, your nerves can’t send and receive signals properly.
- [Giving Tuesday](https://krabbeconnect.org/kcevent/giving-tuesday/) - What is Giving Tuesday? Giving Tuesday is a global day of charitable giving and philanthropy that follows the Thanksgiving holiday weekend. It was created to encourage people around the world to dedicate a day to giving back to their communities and supporting various causes. The main idea behind Giving Tuesday is to counterbalance the consumerism
- [#CureKrabbe Family Conference](https://krabbeconnect.org/kcevent/curekrabbe-family-meeting/) - KrabbeConnect is hosting its first in-person #curekrabbe Family Conference in Tampa Florida, April 21st-23rd, 2023. The family conference will feature scientific, advocacy, and family support sessions. The conference is designed to empower patients and their families and caregivers by providing information and tools to help navigate Krabbe disease. KrabbeConnect wants you to use this time to connect and learn from others walking in your shoes, hear from the medical and research community on recent updates, and get to know the companies supporting Krabbe disease.

## Heroes

- [Slade C.](https://krabbeconnect.org/krabbe-hero/slade-c/) - Wales, MI
- [Mark P.](https://krabbeconnect.org/krabbe-hero/mark-p/) - Grand Rapids, MI
- [Kinsley T](https://krabbeconnect.org/krabbe-hero/kinsley-t/) - Augusta GA
- [Maria Cecília R.](https://krabbeconnect.org/krabbe-hero/maria-cecilia-r/) - Campo Grande, Brazil
- [Weston K](https://krabbeconnect.org/krabbe-hero/weston-k/) - Black River Falls, WI
- [Penelope B.](https://krabbeconnect.org/krabbe-hero/penelope-b/) - NC
- [Nikolas H](https://krabbeconnect.org/krabbe-hero/nikolas-h/) - San Antonio
- [Emmett Y](https://krabbeconnect.org/krabbe-hero/emmett-y/) - Lawrenceburg, IN
- [Saylor O.](https://krabbeconnect.org/krabbe-hero/saylor-o/) - Excelsior, MN
- [Conn M.](https://krabbeconnect.org/krabbe-hero/conn-m/) - Ballymena, Ireland
- [Jackson W.](https://krabbeconnect.org/krabbe-hero/jackson-w/) - Rhome, TX
- [Aron Eli O.](https://krabbeconnect.org/krabbe-hero/aron-eli-o/) - Reykjavik, Iceland
- [Anna T.](https://krabbeconnect.org/krabbe-hero/anna-t/) - Beaver Dam, KY
- [Naomi H.](https://krabbeconnect.org/krabbe-hero/naomi-h/) - Simpsonville, SC
- [Mabry Kate W.](https://krabbeconnect.org/krabbe-hero/mabry-kate-w/) - Powell, TX
- [Lennon F.](https://krabbeconnect.org/krabbe-hero/lennon-f/) - Piketon, OH
- [Owen W.](https://krabbeconnect.org/krabbe-hero/owen-w/) - Powell, TX
- [Blaine S](https://krabbeconnect.org/krabbe-hero/blaine-s/) - Ohio
- [Christopher C.](https://krabbeconnect.org/krabbe-hero/christopher-c-2/) - Marana, AZ
- [Sophie](https://krabbeconnect.org/krabbe-hero/sophie/) - Tyne and Wear uk
- [Cassie D.](https://krabbeconnect.org/krabbe-hero/cassie-d/) - Richmond, Va
- [Charlotte H](https://krabbeconnect.org/krabbe-hero/charlotte-h/) - Fort Collins, CO
- [Lily S](https://krabbeconnect.org/krabbe-hero/lily-s/) - Southern Maryland
- [Josiah H](https://krabbeconnect.org/krabbe-hero/josiah-h/) - Burlington, CO
- [Saylor Scott](https://krabbeconnect.org/krabbe-hero/saylor-scott/) - Kalamazoo, MI
- [Ronan P.](https://krabbeconnect.org/krabbe-hero/ronan-p/) - Columbia, MO
- [Connor W](https://krabbeconnect.org/krabbe-hero/connor-w/) - Coeur d’Alene, ID
- [Jaxon T](https://krabbeconnect.org/krabbe-hero/jaxon-t/) - Augusta GA
- [Cristhiam C](https://krabbeconnect.org/krabbe-hero/cristhiam-c/) - Douglasville, GA
- [Ella Rose F](https://krabbeconnect.org/krabbe-hero/ella-rose-f/) - Rochester, NY
- [Zayn N](https://krabbeconnect.org/krabbe-hero/zayn-n/) - Brisbane, Queensland
- [Louis L](https://krabbeconnect.org/krabbe-hero/louis-l/) - Wollongong, NSW, Australia
- [David Fernández Serrano](https://krabbeconnect.org/krabbe-hero/david-fernandez-serrano/) - Guadalajara jalisco México
- [Jia Kim](https://krabbeconnect.org/krabbe-hero/jia-kim/) - Seoul, Republic of Korea
- [Zoey L](https://krabbeconnect.org/krabbe-hero/zoey-l/) - Olmsted Twsp, Ohio
- [Madison H](https://krabbeconnect.org/krabbe-hero/madison-h/) - OH
- [Логачева София Викторовна](https://krabbeconnect.org/krabbe-hero/логачева-софия-викторовна/) - Днепр
- [Sydney C](https://krabbeconnect.org/krabbe-hero/sydney-c/) - Decatur GA
- [Zion V](https://krabbeconnect.org/krabbe-hero/zion-v-2/) - Minneapolis, Minnesota
- [Tenley Thompson](https://krabbeconnect.org/krabbe-hero/tenley-thompson/) - Pennock, MN
- [Makayla P.](https://krabbeconnect.org/krabbe-hero/makayla-p/) - Burnsville, MN
- [Belle M.](https://krabbeconnect.org/krabbe-hero/belle-m/) - Stillwater, MN
- [Grazia S.](https://krabbeconnect.org/krabbe-hero/grazia-s/) - Siracusa, Italy
- [Daniel H.](https://krabbeconnect.org/krabbe-hero/daniel-h/) - San Jose, CA
- [Nico B.](https://krabbeconnect.org/krabbe-hero/nico-b/) - Rockford, ILRead Family Story
- [McKenna T.](https://krabbeconnect.org/krabbe-hero/mckenna-t/) - Modesto, CA
- [Marcus M.](https://krabbeconnect.org/krabbe-hero/marcus-m/) - Wyandotte, MI
- [Sulaiman M.](https://krabbeconnect.org/krabbe-hero/sulaiman-m/) - Buford, GA
- [Patrick S.](https://krabbeconnect.org/krabbe-hero/patrick-surace/) - Svizzera
- [David A.](https://krabbeconnect.org/krabbe-hero/david-aaron/) - Garner, NC
- [Hristo L.](https://krabbeconnect.org/krabbe-hero/hristo-l/) - Birmingham, UK
- [Dennis M.Ø](https://krabbeconnect.org/krabbe-hero/dennis-m-o/) - Norway
- [Scarlett M.](https://krabbeconnect.org/krabbe-hero/scarlett-m/) - Pikeville, TN
- [Reuben N.](https://krabbeconnect.org/krabbe-hero/reuben-nicholls/) - Dunedin, New Zealand
- [Regann M.](https://krabbeconnect.org/krabbe-hero/regann-moore/) - Springfield, MO
- [Jhyrve S.](https://krabbeconnect.org/krabbe-hero/jhyrve-s/) - Pittsburg, CA
- [Emmett K.](https://krabbeconnect.org/krabbe-hero/emmett/) - Clear Lake, SD
- [Angela A.](https://krabbeconnect.org/krabbe-hero/angela-a/) - San Antonio, TX
- [Angelita D.](https://krabbeconnect.org/krabbe-hero/angelita-d/) - Honduras
- [Anna Rose J.](https://krabbeconnect.org/krabbe-hero/anna-rose-j/) - Bracknell, UK
- [Avery C.](https://krabbeconnect.org/krabbe-hero/avery-c/) - Warwick, RI
- [Aubrey C.](https://krabbeconnect.org/krabbe-hero/aubrey-c/) - Cuyahoga Falls, OH
- [Anniston B.](https://krabbeconnect.org/krabbe-hero/anniston-b/) - Lafayette, LA
- [Brittany W.](https://krabbeconnect.org/krabbe-hero/brittany-w/) - Radcliff, KY
- [Bryce C.](https://krabbeconnect.org/krabbe-hero/bryce-c/) - Indianapolis, IN
- [Bryan W.](https://krabbeconnect.org/krabbe-hero/bryan-w/) - Hodgenville, KY
- [Cade H.](https://krabbeconnect.org/krabbe-hero/cade-h/) - Calgary, AB
- [Casey M.](https://krabbeconnect.org/krabbe-hero/casey-m/) - Ireland
- [Cassidy J.](https://krabbeconnect.org/krabbe-hero/cassidy-j/) - Montevideo, MN
- [Christopher S.](https://krabbeconnect.org/krabbe-hero/christopher-s/) - Metolius, OR
- [Darian H.](https://krabbeconnect.org/krabbe-hero/darian-h/) - Humacao, Puerto Rico
- [Cloud K.](https://krabbeconnect.org/krabbe-hero/cloud-k/) - Bainbridge, GA
- [Collin C.](https://krabbeconnect.org/krabbe-hero/collin-c/) - Wisconsin Rapids, WI
- [David B.](https://krabbeconnect.org/krabbe-hero/david-b/) - Akron, OH
- [Dawson F.](https://krabbeconnect.org/krabbe-hero/dawson-f/) - Franklin, VA
- [Delilah S.](https://krabbeconnect.org/krabbe-hero/delilah-s/) - Kent, UK
- [Duarte E.](https://krabbeconnect.org/krabbe-hero/duarte-e/) - Braga, Portugal
- [Dylan M.](https://krabbeconnect.org/krabbe-hero/dylan-m/) - Washington, IL
- [Jayven R.](https://krabbeconnect.org/krabbe-hero/jayven-r/) - Bay St. Louis, MS
- [Jacksea T.](https://krabbeconnect.org/krabbe-hero/jacksea-t/) - Grand Forks, ND
- [Grayson M.](https://krabbeconnect.org/krabbe-hero/grayson-m/) - Boise, ID
- [Emmett B.](https://krabbeconnect.org/krabbe-hero/emmett-b/) - Harrisburg, NC
- [Emmett M.](https://krabbeconnect.org/krabbe-hero/emmett-m/) - Beaverton, OR
- [Evie Beth S.](https://krabbeconnect.org/krabbe-hero/evie-beth-s/) - North Yorkshire, UK
- [Ezra B.](https://krabbeconnect.org/krabbe-hero/ezra-b/) - St. Louis, MO
- [Ginevra A.](https://krabbeconnect.org/krabbe-hero/ginevra-a/) - Alessandria, Italy
- [Jeramie E.](https://krabbeconnect.org/krabbe-hero/jeramie-e/) - Janesville, WI
- [Jody V.](https://krabbeconnect.org/krabbe-hero/jody-v/) - N. Liberty, IA
- [Joshua B.](https://krabbeconnect.org/krabbe-hero/joshua-b/) - New Milford, PA
- [Joshua C.](https://krabbeconnect.org/krabbe-hero/joshua-c/) - Cedar Rapids, IA
- [Juni H.](https://krabbeconnect.org/krabbe-hero/juni-h/) - Gothenburg, Sweden
- [Kenan W.](https://krabbeconnect.org/krabbe-hero/kenan-w/) - Chicago, IL
- [Kian and Luis K.](https://krabbeconnect.org/krabbe-hero/kian-and-luis-k/) - UK
- [Kyran D.](https://krabbeconnect.org/krabbe-hero/kyran-d/) - Brisbane, Australia
- [Lana S.](https://krabbeconnect.org/krabbe-hero/lana-s/) - North Riverside, IL
- [Laura C.](https://krabbeconnect.org/krabbe-hero/laura-c/) - Cedar Rapids, IA
- [Levi P.](https://krabbeconnect.org/krabbe-hero/levi-p/) - Ontario, Canada
- [Lucas N.](https://krabbeconnect.org/krabbe-hero/lucas-n/) - Pensacola, FL
- [Marcus G.](https://krabbeconnect.org/krabbe-hero/marcus-g/) - La Pryor, TX
- [Maria E.](https://krabbeconnect.org/krabbe-hero/maria-e/) - Barcelos, Portugal
- [Mariah G.](https://krabbeconnect.org/krabbe-hero/mariah-g/) - La Pryor, TX
- [Marshall W.](https://krabbeconnect.org/krabbe-hero/marshall-w/) - Dayton, OR
- [Martin S.](https://krabbeconnect.org/krabbe-hero/martin-s/) - Exeter, UK
- [Matthew S.](https://krabbeconnect.org/krabbe-hero/matthew-s/) - Clare, MI
- [Max B.](https://krabbeconnect.org/krabbe-hero/max-b/) - Phoenix, AZ
- [Michael W.](https://krabbeconnect.org/krabbe-hero/michael-w/) - Dayton, OR
- [Mila L.](https://krabbeconnect.org/krabbe-hero/mila-l/) - Southaven, MS
- [Nassiah J.](https://krabbeconnect.org/krabbe-hero/nassiah-j/) - Chicago, IL
- [Nicholas B.](https://krabbeconnect.org/krabbe-hero/nicholas-b/) - New Milford, PA
- [Nick R.](https://krabbeconnect.org/krabbe-hero/nick-r/) - Cincinnati, OH
- [Nikola G.](https://krabbeconnect.org/krabbe-hero/nikola-g/) - Newport News, VA
- [Odin FW.](https://krabbeconnect.org/krabbe-hero/odin-fw/) - Johnson City, NY
- [Oliver P.](https://krabbeconnect.org/krabbe-hero/oliver-p/) - Surrey, UK
- [Taiten B.](https://krabbeconnect.org/krabbe-hero/taiten-b/) - Eagle Mountain, UT
- [Sunny H.](https://krabbeconnect.org/krabbe-hero/sunny-h/) - Bayside, CA
- [Sophia S.](https://krabbeconnect.org/krabbe-hero/sophia-s/) - Kannapolis, NC
- [Quinton N.](https://krabbeconnect.org/krabbe-hero/quinton-n/) - Des Plaines, IL
- [Parker Janelle G.](https://krabbeconnect.org/krabbe-hero/parker-janelle-g/) - Tempe, AZ
- [Rebekah F.](https://krabbeconnect.org/krabbe-hero/rebekah-f/) - Western Cape, South Africa
- [Reesa S.](https://krabbeconnect.org/krabbe-hero/reesa-s/) - Scottsdale, AZ
- [Rocco W.](https://krabbeconnect.org/krabbe-hero/rocco-w/) - London, UK
- [Rowan S.](https://krabbeconnect.org/krabbe-hero/rowan-s/) - Nova Scotia, Canada
- [Sandrine D.](https://krabbeconnect.org/krabbe-hero/sandrine-sd/) - Montreal, Canada
- [Thomas H.](https://krabbeconnect.org/krabbe-hero/thomas-h/) - Ayden, NC
- [Tygh D.](https://krabbeconnect.org/krabbe-hero/tygh-d/) - Puryear, TN
- [Victoria B.](https://krabbeconnect.org/krabbe-hero/victoria-b/) - Hershey, PA
- [Tymoteusz Mikołaj K.](https://krabbeconnect.org/krabbe-hero/tymoteusz-mikolaj-k/) - Pław, Poland
- [Vladimir D.](https://krabbeconnect.org/krabbe-hero/vladimir-d/) - Comayagua, Honduras
- [William B.](https://krabbeconnect.org/krabbe-hero/william-b/) - Davenport, IA
- [Zander M.](https://krabbeconnect.org/krabbe-hero/zander-m/) - Erie, PA
- [Artëm D.](https://krabbeconnect.org/krabbe-hero/artem-d/) - r.p.Sargatskoe Omskaja oblast
- [Alessandro G.](https://krabbeconnect.org/krabbe-hero/alessandro-g/) - Roma, Italia
- [Jasper M.](https://krabbeconnect.org/krabbe-hero/jasper-m/) - Columbus, OH
- [Adam H.](https://krabbeconnect.org/krabbe-hero/adam-h/) - Maple Grove, MN
- [Zoey M.](https://krabbeconnect.org/krabbe-hero/zoey-m/) - Atlantic, IA
- [Raegan M.](https://krabbeconnect.org/krabbe-hero/raegan-m/) - York, NE
- [Jaydev L.](https://krabbeconnect.org/krabbe-hero/jaydev-lalu/) - Jetpur Gujarat, India
- [Elijah B.](https://krabbeconnect.org/krabbe-hero/elijah-b/) - Chicago, IL
- [Alissa B.](https://krabbeconnect.org/krabbe-hero/alissa-b/) - Chicago, IL
- [Bentley S.](https://krabbeconnect.org/krabbe-hero/bentley-s/) - Mayodan, NC
- [Leandra F.](https://krabbeconnect.org/krabbe-hero/leandra-f/) - Brazil, IN
- [Ashley C.](https://krabbeconnect.org/krabbe-hero/ashley-c/) - Sun Prairire, WI
- [Jeremy T.](https://krabbeconnect.org/krabbe-hero/jeremy-t/) - Eau Claire, WI
- [Trevor L.](https://krabbeconnect.org/krabbe-hero/trevor-l/) - Baldwin City, KS
- [Liam H.](https://krabbeconnect.org/krabbe-hero/liam-h/) - Chicago, IL
- [Julia B.](https://krabbeconnect.org/krabbe-hero/julia-b/) - Ann Arbor, MI
- [Jackson G.](https://krabbeconnect.org/krabbe-hero/jackson-g/) - Lawrenceville, GA
- [Hasan M.](https://krabbeconnect.org/krabbe-hero/hasan-m/) - Melrose, MA
- [Gina R.](https://krabbeconnect.org/krabbe-hero/gina-r/) - Cincinatti, OH
- [Anthony S.](https://krabbeconnect.org/krabbe-hero/anthony-s/) - Millers Creek, NC
- [Anthony C.](https://krabbeconnect.org/krabbe-hero/anthony-c/) - Brooklyn, NY
- [Alex T.](https://krabbeconnect.org/krabbe-hero/alex-t-2/) - Eau Claire, WI
- [Dalton S.](https://krabbeconnect.org/krabbe-hero/dalton-s-2/) - Millers Creek, NC
- [Lucille F.](https://krabbeconnect.org/krabbe-hero/lucille-f/) - Ozone, AR
- [Kohen K.](https://krabbeconnect.org/krabbe-hero/kohen-k/) - Pekin, IL
- [Graham C.](https://krabbeconnect.org/krabbe-hero/graham-c/) - Morton, IL
- [Kody G.](https://krabbeconnect.org/krabbe-hero/kody-g/) - Caldwell, ID
- [Mason L.](https://krabbeconnect.org/krabbe-hero/mason-l/) - Selangor, Malaysia
- [Eva-Rose S.](https://krabbeconnect.org/krabbe-hero/eva-rose-s/) - Derby, United Kingdom
- [Jace P.](https://krabbeconnect.org/krabbe-hero/jace-p/) - Portland, MI
- [Anthony M.](https://krabbeconnect.org/krabbe-hero/anthony-m/) - Wyandotte, MI
- [Giovanni M.](https://krabbeconnect.org/krabbe-hero/giovanni-m/) - Lakewood, OH
- [Jaxon S.](https://krabbeconnect.org/krabbe-hero/jaxon-s/) - Galesburg, IL
- [Elif C.](https://krabbeconnect.org/krabbe-hero/elif-c/) - Istanbul, Turkey
- [Emmalynn A.](https://krabbeconnect.org/krabbe-hero/emmalynn-a/) - Neon, KY
- [DJ O.](https://krabbeconnect.org/krabbe-hero/damon-james-dj/) - Havertown, PA
- [Sophie T.](https://krabbeconnect.org/krabbe-hero/sophie-t/) - Gateshead, UK
- [Roman  R.](https://krabbeconnect.org/krabbe-hero/roman-r/) - Poole dorset, UK
- [Sakina M.](https://krabbeconnect.org/krabbe-hero/sakina-m/) - Dawsonville GA
- [Alexander L.](https://krabbeconnect.org/krabbe-hero/alexander-l/) - Morgantown, WV

## States

- [Newborn Screening for Krabbe Disease in Arkansas](https://krabbeconnect.org/states/ar/) - DOWNLOAD STATE FACT SHEET AS PDF In 2020, there were 35,251 live births in Arkansas2 There are 33 conditions on the Arkansas Newborn Screening Panel3 All babies born in the United States are screened for several conditions shortly after birth. Approximately 24-48 hours after a baby is born in the United States, a nurse pricks
- [California](https://krabbeconnect.org/states/ca/) - A Fact Sheet about NBS for the state you've chosen is coming soon.
- [Newborn Screening for Krabbe Disease in Arizona](https://krabbeconnect.org/states/az/) - DOWNLOAD STATE FACT SHEET AS PDF In 2020, there were 76,947 live births in Arizona2 There are 33 conditions on the Arizona Newborn Screening Panel3 All babies in the United States are screened for several conditions shortly after birth. Approximately 24-48 hours after a baby is born in the United States, the heel is pricked
- [Newborn Screening for Krabbe Disease in Nebraska](https://krabbeconnect.org/states/ne/) - DOWNLOAD STATE FACT SHEET AS PDF In 2020, there were 24,291 live births in Nebraska2 There are 35 conditions on the Nebraska Newborn Screening Panel3 All babies in the United States are screened for several conditions shortly after birth. Approximately 24-48 hours after a baby is born in the United States, the heel is pricked
- [Mississippi](https://krabbeconnect.org/states/ms/) - A Fact Sheet about NBS for the state you've chosen is coming soon.
- [Newborn Screening for Krabbe Disease in North Carolina](https://krabbeconnect.org/states/nc/) - DOWNLOAD STATE FACT SHEET AS PDF In 2020, there were 116,730 live births in North Carolina2 There are 41 conditions on the North Carolina Newborn Screening Panel3 All babies in the United States are screened for several conditions shortly after birth. Approximately 24-48 hours after a baby is born in the United States, the heel
- [Wisconsin](https://krabbeconnect.org/states/wi/) - A Fact Sheet about NBS for the state you've chosen is coming soon.
- [Rhode Island](https://krabbeconnect.org/states/ri/) - A Fact Sheet about NBS for the state you've chosen is coming soon.
- [Newborn Screening for Krabbe Disease in Massachusetts](https://krabbeconnect.org/states/ma/) - DOWNLOAD STATE FACT SHEET AS PDF In 2020, there were 66,428 live births in Massachusetts2 There are 66 conditions on the Massachusetts Newborn Screening Panel3 All babies in the United States are screened for several conditions shortly after birth. Approximately 24-48 hours after a baby is born in the United States, the heel is pricked
- [Maine](https://krabbeconnect.org/states/me/) - A Fact Sheet about NBS for the state you've chosen is coming soon.
- [New Hampshire](https://krabbeconnect.org/states/nh/) - A Fact Sheet about NBS for the state you've chosen is coming soon.
- [Vermont](https://krabbeconnect.org/states/vt/) - A Fact Sheet about NBS for the state you've chosen is coming soon.
- [Newborn Screening for Krabbe Disease in Utah](https://krabbeconnect.org/states/ut/) - DOWNLOAD STATE FACT SHEET AS PDF In 2020, there were 45,702 live births in Utah2 There are 53 conditions on the Utah Newborn Screening Panel3 All babies born in the United States are screened for several conditions shortly after birth. Approximately 24-48 hours after a baby is born in the United States, a nurse pricks
- [North Dakota](https://krabbeconnect.org/states/nd/) - A Fact Sheet about NBS for the state you've chosen is coming soon.
- [New Mexico](https://krabbeconnect.org/states/nm/) - A Fact Sheet about NBS for the state you've chosen is coming soon.
- [Newborn Screening for Krabbe Disease in Oregon](https://krabbeconnect.org/states/or/) - DOWNLOAD STATE FACT SHEET AS PDF In 2020, there were 39,820 live births in Oregon2 There are 42 conditions on the Oregon Newborn Screening Panel3 All babies in the United States are screened for several conditions shortly after birth. Approximately 24-48 hours after a baby is born in the United States, the heel is pricked
- [Newborn Screening for Krabbe Disease in Kansas](https://krabbeconnect.org/states/ks/) - DOWNLOAD STATE FACT SHEET AS PDF In 2020, there were 34,376 live births in Kansas2 There are 34 conditions on the Kansas Newborn Screening Panel3 All babies in the United States are screened for several conditions shortly after birth. Approximately 24-48 hours after a baby is born in the United States, the heel is pricked
- [Newborn Screening for Krabbe Disease in Maryland](https://krabbeconnect.org/states/md/) - DOWNLOAD STATE FACT SHEET AS PDF In 2020, there were 68,554 live births in Maryland2 There are 61 conditions on the Maryland Newborn Screening Panel3 All babies in the United States are screened for several conditions shortly after birth. Approximately 24-48 hours after a baby is born in the United States, the heel is pricked
- [Newborn Screening for Krabbe Disease in Iowa](https://krabbeconnect.org/states/ia/) - DOWNLOAD STATE FACT SHEET AS PDF In 2020, there were 36,114 live births in Iowa2 There are 53 conditions on the Iowa Newborn Screening Panel3 All babies in the United States are screened for several conditions shortly after birth. Approximately 24-48 hours after a baby is born in the United States, a nurse pricks the
- [Texas](https://krabbeconnect.org/states/tx/) - A Fact Sheet about NBS for the state you've chosen is coming soon.
- [Alabama](https://krabbeconnect.org/states/al/) - A Fact Sheet about NBS for the state you've chosen is coming soon.
- [Newborn Screening for Krabbe Disease in Minnesota](https://krabbeconnect.org/states/mn/) - DOWNLOAD STATE FACT SHEET AS PDF In 2020, there were 63,443 live births in Minnesota2 There are 61 conditions on the Minnesota Newborn Screening Panel 3 All babies in the United States are screened for several conditions shortly after birth. Approximately 24-48 hours after a baby is born in the United States, the heel is
- [Newborn Screening for Krabbe Disease in New Jersey](https://krabbeconnect.org/states/nj/) - DOWNLOAD STATE FACT SHEET AS PDF In 2020, there were 97,954 live births in New Jersey2 There are 62 conditions on the New Jersey Newborn Screening Panel 3 All babies in the United States are screened for several conditions shortly after birth. Approximately 24-48 hours after a baby is born in the United States, the
- [Wyoming](https://krabbeconnect.org/states/wy/) - A Fact Sheet about NBS for the state you've chosen is coming soon.
- [West Virginia](https://krabbeconnect.org/states/wv/) - A Fact Sheet about NBS for the state you've chosen is coming soon.
- [Washington](https://krabbeconnect.org/states/wa/) - A Fact Sheet about NBS for the state you've chosen is coming soon.
- [Newborn Screening for Krabbe Disease in South Dakota](https://krabbeconnect.org/states/sd/) - DOWNLOAD STATE FACT SHEET AS PDF In 2020, there were 10,960 live births in South Dakota2 There are 50 conditions on the South Dakota Newborn Screening Panel3 All babies born in the United States are screened for several conditions shortly after birth. Approximately 24-48 hours after a baby is held in the United States, a
- [Oklahoma](https://krabbeconnect.org/states/ok/) - A Fact Sheet about NBS for the state you've chosen is coming soon.
- [Nevada](https://krabbeconnect.org/states/nv/) - A Fact Sheet about NBS for the state you've chosen is coming soon.
- [Montana](https://krabbeconnect.org/states/mt/) - A Fact Sheet about NBS for the state you've chosen is coming soon.
- [Newborn Screening for Krabbe Disease in Idaho](https://krabbeconnect.org/states/id/) - DOWNLOAD STATE FACT SHEET AS PDF In 2020, there were 21,533 live births in Idaho2 There are 52 conditions on the Idaho Newborn Screening Panel3 All babies in the United States are screened for several conditions shortly after birth. Approximately 24-48 hours after a baby is born in the United States, the heel is pricked
- [Hawaii](https://krabbeconnect.org/states/hi/) - A Fact Sheet about NBS for the state you've chosen is coming soon.
- [Connecticut](https://krabbeconnect.org/states/ct/) - A Fact Sheet about NBS for the state you've chosen is coming soon.
- [Colorado](https://krabbeconnect.org/states/co/) - A Fact Sheet about NBS for the state you've chosen is coming soon.
- [Alaska](https://krabbeconnect.org/states/ak/) - A Fact Sheet about NBS for the state you've chosen is coming soon.
- [Newborn Screening for Krabbe Disease in Michigan](https://krabbeconnect.org/states/mi/) - DOWNLOAD STATE FACT SHEET AS PDF In 2020, there were 104,074 live births in Michigan2 There are 58 conditions on the Michigan Newborn Screening Panel3 All babies in the United States are screened for several conditions shortly after birth. Approximately 24-48 hours after a baby is born in the United States, the heel is pricked
- [Newborn Screening for Krabbe Disease in Georgia](https://krabbeconnect.org/states/ga/) - DOWNLOAD STATE FACT SHEET AS PDF In 2020, there were 122,473 live births in Georgia 2 There are 33 conditions on the Georgia Newborn Screening Panel 3 All babies in the United States are screened for several conditions shortly after birth. Approximately 24-48 hours after a baby is born in the United States, the heel
- [Newborn Screening for Krabbe Disease in Tennessee](https://krabbeconnect.org/states/tn/) - DOWNLOAD STATE FACT SHEET AS PDF In 2020, there were 78,689 live births in Tennessee2 There are 71 conditions on the Tennessee Newborn Screening Panel3 All babies in the United States are screened for several conditions shortly after birth. Approximately 24-48 hours after a baby is born in the United States, the heel is pricked
- [Newborn Screening for Krabbe Disease in Kentucky](https://krabbeconnect.org/states/ky/) - DOWNLOAD STATE FACT SHEET AS PDF In 2020, there were 51,668 live births in Kentucky 2 There are 59 conditions on the Kentucky Newborn Screening Panel 3 All babies in the United States are screened for several conditions shortly after birth. Approximately 24-48 hours after a baby is born in the United States, the heel
- [Delaware](https://krabbeconnect.org/states/de/) - A Fact Sheet about NBS for the state you've chosen is coming soon.
- [New York](https://krabbeconnect.org/states/ny/) - A Fact Sheet about NBS for the state you've chosen is coming soon.
- [Newborn Screening for Krabbe Disease in Pennsylvania](https://krabbeconnect.org/states/pa/) - DOWNLOAD STATE FACT SHEET AS PDF In 2020, there were 130,693 live births in Pennsylvania2 There are 61 conditions on the Pennsylvania Newborn Screening Panel3 All babies in the United States are screened for several conditions shortly after birth. Approximately 24-48 hours after a baby is born in the United States, a nurse pricks the
- [Newborn Screening for Krabbe Disease in Ohio](https://krabbeconnect.org/states/oh/) - DOWNLOAD STATE FACT SHEET AS PDF In 2020, there were 129,191 live births in Ohio2 There were 8 infants diagnosed with Krabbe disease since 2016 All babies in the United States are screened for several conditions shortly after birth. Approximately 24-48 hours after a baby is born in the United States, the heel is pricked
- [Newborn Screening for Krabbe Disease in Indiana](https://krabbeconnect.org/states/in/) - DOWNLOAD STATE FACT SHEET AS PDF In 2020, there were 78,616 live births in Indiana2 There are 58 conditions on the Indiana Newborn Screening Panel3 All babies born in the United States are screened for several conditions shortly after birth. Approximately 24-48 hours after a baby is born in the United States, a nurse pricks
- [Newborn Screening for Krabbe Disease in Illinois](https://krabbeconnect.org/states/il/) - DOWNLOAD STATE FACT SHEET AS PDF In 2020, there were 133,298 live births in Illinois2 There are 57 conditions on the Illinois Newborn Screening Panel3 All babies in the United States are screened for several conditions shortly after birth. Approximately 24-48 hours after a baby is born in the United States, the heel is pricked
- [Newborn Screening for Krabbe Disease in Missouri](https://krabbeconnect.org/states/mo/) - DOWNLOAD STATE FACT SHEET AS PDF In 2020, there were 69,285 live births in Missouri2 There are 77 conditions on the Missouri Newborn Screening Panel3 All babies born in the United States are screened for several conditions shortly after birth. Approximately 24-48 hours after a baby is born in the United States, a nurse pricks
- [South Carolina](https://krabbeconnect.org/states/sc/) - A Fact Sheet about NBS for the state you've chosen is coming soon.
- [Newborn Screening for Krabbe Disease in Florida](https://krabbeconnect.org/states/fl/) - DOWNLOAD STATE FACT SHEET AS PDF In 2020, there were 209,671 live births in Florida2 There are 57 conditions on the Florida Newborn Screening Panel3 All babies in the United States are screened for several conditions shortly after birth. Approximately 24-48 hours after a baby is born in the United States, the heel is pricked
- [Newborn Screening for Krabbe Disease in Louisiana](https://krabbeconnect.org/states/la/) - DOWNLOAD STATE FACT SHEET AS PDF In 2020, there were 57,328 live births in Louisiana2 There are 34 conditions on the Louisiana Newborn Screening Panel3 All babies in the United States are screened for several conditions shortly after birth. Approximately 24-48 hours after a baby is born in the United States, the heel is pricked
- [Newborn Screening for Krabbe Disease in Virginia](https://krabbeconnect.org/states/va/) - DOWNLOAD STATE FACT SHEET AS PDF In 2020, there were 94,749 live births in Virginia 2 There are 35 conditions on the Virginia Newborn Screening Panel 3 All babies in the United States are screened for several conditions shortly after birth. Approximately 24-48 hours after a baby is born in the United States, the heel

## YouTube Videos

- [Grief, Love, and the Holidays: Creating Space for What We Carry](https://krabbeconnect.org/videos/grief-love-and-the-holidays-creating-space-for-what-we-carry/) - The holidays can be challenging, especially when you’re carrying the weight of caregiving or the grief of losing a child to Krabbe disease. Sometimes, you just need a safe space to acknowledge those feelings and talk with others who truly understand. Watch this 20-minute conversation with Sarah Thomas, MSW, LICSW—a video created to offer comfort,
- [Insights and Data from the ReKlaim (FBX-101) Clinical Trial](https://krabbeconnect.org/videos/insights-and-data-from-the-reklaim-fbx-101-clinical-trial/) - About the Webinar (Recorded on September 26th, 2024) This session will provide an in-depth look at the latest findings from these pivotal trials focused on treatments for Krabbe disease. Attendees will gain valuable knowledge on trial outcomes, patient responses, and the potential implications for future therapies. Whether you’re a researcher, clinician, or someone impacted by
- [Caregiver Webinar: Experiences Navigating Treatment for Krabbe disease](https://krabbeconnect.org/videos/caregiver-webinar-experiences-navigating-treatment-for-krabbe-disease/) - This panel was recorded on September 16th, 2024. The panel, moderated by Stacy Pike-Langenfeld, explores various experiences, showcasing the challenges and triumphs in the treatment of Krabbe disease. Each caregiver will talk about their child’s journey. Each journey is unique, giving healthcare providers and newly diagnosed families "insider knowledge," of what that first year could
- [IKD and LIKD Study](https://krabbeconnect.org/videos/ikd-and-likd-study/) - A Krabbe disease study led by Dr. Nicholas Bascou. Dr. Bascou completed his undergraduate studies at the University of Pittsburgh at The Program for the Study of Neurodevelopment in Rare Disorders (NDRD). He then obtained his M.D. at the Cooper Medical School of Rowan University in Camden, New Jersey, and completed his training in internal
- [Two Sisters Journey with Krabbe disease:  The Moore Family Story](https://krabbeconnect.org/videos/two-sisters-journey-with-krabbe-disease-the-moore-family-story/) - Meet the Moore sisters. Raegan missed the window of opportunity for a potentially life-saving intervention because her formal diagnosis did not arrive until she was 9 months old. Raegan faced the heartbreaking challenges of severe neurological deterioration, loss of mobility, and a shortened life expectancy. Meet Zoey, a joyful and energetic young adult fortunate to
- [(Uncut interview) How Grandpa Bruce Reworks Cove's Law in Georgia](https://krabbeconnect.org/videos/uncut-interview-how-grandpa-bruce-reworks-coves-law-in-georgia/) - This is the full heartwarming interview with Bruce Kirbo. Grandpa Bruce went to work on Cove's Law after his grandson, Cloud, was diagnosed with Krabbe disease. Listen to how Bruce, and other advocates, successfully launched a pilot study in Georgia for Krabbe disease. Krabbe disease (pronounced krab A) is a rare genetic disorder known as
- [Cloud Family Interview (uncut)](https://krabbeconnect.org/videos/cloud-family-interview-uncut/) - This is the uncut video from our interview with the Kirbo family. Learn about all of the emotions involved in navigating Krabbe disease through the eyes of the Kirbo family. KrabbeConnect is forever grateful to Carlyle and Sloane for sharing their son's story to help further education and improve awareness about the need for additional
- [Grandpa Bruce Reworks Cove's Law in Georgia Final Draft](https://krabbeconnect.org/videos/grandpa-bruce-reworks-coves-law-in-georgia-final-draft/) - Bruce Kirbo went to work on Cove's Law after his grandson, Cloud, was diagnosed with Krabbe disease. Listen to how Bruce, and other advocates, successfully launched a pilot study in Georgia for Krabbe disease. Krabbe disease (pronounced krab A), is a rare genetic disorder, also known as globoid cell leukodystrophy. Krabbe disease is a severe
- [How the Kirbo and Ellis Family Navigate Krabbe disease](https://krabbeconnect.org/videos/how-the-kirbo-and-ellis-family-navigate-krabbe-disease/) - We invite you to learn about the Kirbo and Ellis families. Both have been impacted by Krabbe disease. Cloud has a late-onset form of Krabbe disease and Cove has the infantile form of the disease. Cloud and Cove offer so much to their families despite their disabilities. Both children are surrounded by love and their
- [KrabbeConnect Presented the 2022 Wise Scholar Award to Dr. Joanne Kurtzberg](https://krabbeconnect.org/videos/krabbeconnect-presented-the-2022-wise-scholar-award-to-dr-joanne-kurtzberg/) - There’s no shortage of talent in the science and clinical space of Krabbe disease. KrabbeConnect believes it’s vital to honor an individual every year who has made a significant contribution(s) to Krabbe disease in one or more of the following areas: educating and improving methods to effectively recognize and diagnose Krabbe disease, advancing breakthrough research
- [#curekrabbe Challenge 2022 updated](https://krabbeconnect.org/videos/curekrabbe-challenge-2022-updated/) - Are you ready for the #curekrabbbe challenge? The Krabbe community of patients and their caregivers are challenged each day due to their Krabbe disease diagnosis. KrabbeConnect works to help families with these challenges by advancing research, providing family support, enhancing education, and raising awareness. KrabbeConnect believes in working together to help assure a better life
- [Newborn Screening Gives Jasper a Chance at Life](https://krabbeconnect.org/videos/newborn-screening-gives-jasper-a-chance-at-life/) - This video debuted at the KrabbeConnect 2021, A Million Dreams Gala. Learn about Jasper who was in foster care, was identified with Krabbe disease as a newborn, and how he's doing today. The Murphy family is thankful for the love and joy Jasper brings to their home.
- [Dr. Allyson Bradbury Pivotal Work in Krabbe disease](https://krabbeconnect.org/videos/dr-allyson-bradbury-pivotal-work-in-krabbe-disease/) - This video was first debuted at the 2021 KrabbeConnect, "A Million Dreams," gala. Dr. Allison Bradbury has evaluated disease mechanisms and developed gene therapy approaches for Krabbe disease. The Bradbury Laboratory is dedicated to understanding disease mechanisms and developing therapeutic approaches for rare, pediatric neurodegenerative disorders. Advances in adeno-associated virus (AAV) vectors have led to
- [Webinar:  Rationale for Reduced-Intensity Conditioning (RIC) Transplantation in Krabbe Disease](https://krabbeconnect.org/videos/webinar-rationale-for-reduced-intensity-conditioning-ric-transplantation-in-krabbe-disease/) - one marrow transplants are utilized in patients diagnosed with Globoid Cell Leukodystrophy (Krabbe) to help treat the debilitating disease. However, some of the chemotherapy medicines utilized to suppress the immune response can leave a patient managing troublesome side effects. Thus, transplant specialists are pioneering a new way to conduct bone marrow transplants in patients with
- [2021 A Million Dreams Gala](https://krabbeconnect.org/videos/2021-a-million-dreams-gala/) - Did you miss the gala on Friday, September 24th, 2021? No problem, you can view it here now. KrabbeConnect is truly thankful to be here, providing those directly living with Krabbe disease educational resources to make difficult decisions a bit easier, connecting them to rare disease experts, and participating in research efforts to help #curekrabbe.
- [KrabbeConnect Gala 2020 Short Interview with Grujicic Family](https://krabbeconnect.org/videos/krabbeconnect-gala-2020-short-interview-with-grujicic-family/) - This video was utilized for the 2020, "A Million Dreams," Gala. A great story of a family impacted by infantile Krabbe disease. If this story moves you, help us reach a cure by donating at KrabbeConnect.org today.
- [Krabbe disease Tribute](https://krabbeconnect.org/videos/krabbe-disease-tribute/) - A one-of-a-kind tribute to our heroes on KrabbeConnect.org. These are the faces of patients living and no longer living with Globoid Cell Leukodystrophy (Krabbe disease). This video closed out the 2020 A Million Dreams Gala to remind our community that we need to continue the fight until we #curekrabbe. Learn more about this disease at
- [Transplant Alone is Not a Cure](https://krabbeconnect.org/videos/transplant-alone-is-not-a-cure/) - A 3-minute video with Krabbe disease expert, Dr. Joanne Kurtzberg, and 4 amazing transplanted Krabbe disease patients. We're so thankful for transplant to help patients with this rare neurological disease live a better life. Yet, it's important we share this video as transplanted patients need additional therapies to ensure they can continue living their best
- [Nikola's Krabbe Disease Story: Extended Interview](https://krabbeconnect.org/videos/nikolas-krabbe-disease-story-extended-interview/) - Dragon and Lana, parents of Nikola, sit down with KrabbeConnect, to share their Krabbe disease journey. Krabbe disease is a rare neurological disease that results from the loss of the protective covering (myelin sheath) surrounding nerve cells. Dragon and Lana, residents of Virginia, share their story with the hopes of further educating the medical community
- ["A Million Dreams" Gala](https://krabbeconnect.org/videos/a-million-dreams-gala/) - Please join us live on October 10th, 2020 for our, "A Million Dreams," gala as KrabbeConnect highlights patient stories, embraces the generosity of our corporate sponsors, pays tribute to those patients diagnosed with Krabbe disease, and fundraises to the max to reach a day in which each patient can live a life free of Krabbe
- [Krabbe Disease: An Autosomal Recessive Disease](https://krabbeconnect.org/videos/krabbe-disease-an-autosomal-recessive-disease/) - Check out this video to learn about Krabbe disease and how autosomal recessive diseases affect people.
- [Krabbe disease: One Parent's Journey](https://krabbeconnect.org/videos/krabbe-disease-one-parents-journey/) - Do you wonder what it's like to care for a child diagnosed with infantile Krabbe disease? Check out this 6-minute view and visit krabbeconnect.org to learn how you can help families impacted by Krabbe disease.
- [Partner With Us   KrabbeConnect final video](https://krabbeconnect.org/videos/partner-with-us-krabbeconnect-final-video/) - The opportunity for a family or an individual to establish a fund, utilizing KrabbeConnect’s non-profit platform and resources, to strengthen groundbreaking research, contribute towards a special project, or simply to aid other impactful efforts in the Krabbe disease space. We encourage you to visit https://krabbeconnect.org/community-engagement/partner-with-krabbe-connect/ to learn more.

## Glossary Terms

- [GALC gene](https://krabbeconnect.org/glossary/galc-gene/) - The GALC gene is located on chromosome 14. This gene provides instructions for making the enzyme galactocerebrosidase. This enzyme breaks down certain fats called galactolipids. Galactocerebrosidase plays a key role in the production of myelin, the protective coating surrounding nerves.
- [Galactocerebrosidase](https://krabbeconnect.org/glossary/galactocerebrosidase/) - A specific enzyme that receives its instructions from the GALC gene. When the GALC gene isn’t working properly, the enzyme, galactocerebrosidase, is unable to break down certain fats called galactolipids. Galactocerebrosidase plays a key role in the production of myelin, the protective coating surrounding nerves.
- [reduced condition](https://krabbeconnect.org/glossary/reduced-condition/) - In traditional transplantation cases, high doses of chemotherapy medications are used. A reduce-conditioning regimen refers to the chemotherapy protocol that patients undergo prior to “transplant day.” The amount of chemotherapy medications is often less than the traditional protocol.
- [Virus Vector](https://krabbeconnect.org/glossary/virus-vector/) - When we think of a virus, we think of getting sick; however, it might surprise you to learn that viruses have specialized mechanisms that help scientists deliver genetic material into cells. There are many types of viral vectors used to advance gene therapy and many more are being discovered.
- [UCBT](https://krabbeconnect.org/glossary/ucbt/) - An umbilical cord blood transplant utilizes blood from the umbilical cord for transplantation purposes. Like bone marrow and peripheral blood, the umbilical cord is also a rich source of stem cells used in transplantation.
- [Stem Cell Transplant](https://krabbeconnect.org/glossary/stem-cell-transplant/) - Typically this refers to a bone marrow transplant with the goal of replacing non-working cells with healthy working cells. Bone marrow is a rich source of stem cells that have the unique ability to divide over and over to provide new cells and as they divide, they have the ability to change into other types
- [Sphingolipidosis](https://krabbeconnect.org/glossary/sphingolipidosis/) - A class of disorders of sphingolipd metabolism, like Krabbe disease, which have an impact on neural tissues in the brain.
- [Spasticity](https://krabbeconnect.org/glossary/spasticity/) - A term that describes muscles that are continually contracted and often described by patients as stiff and tight muscles. This stiffness and tightness can interfere with many activities of daily living like walking and even talking. Spasticity is typically caused by damage to certain areas of the brain caused by Krabbe disease.
- [Schwann Cells](https://krabbeconnect.org/glossary/schwann-cells/) - Schwann cells are involved in many important aspects of the peripheral nervous system (PNS) that produce the myelin sheath (protective coating) surrounding nerves. Schwann cells are akin to the peripheral nervous system as oligodentrocytes are to the central nervous system.
- [Peripheral Nervous System (PNS)](https://krabbeconnect.org/glossary/peripheral-nervous-system-pns/) - Part of the nervous system that resides outside the brain and spinal cord.
- [Psychosine](https://krabbeconnect.org/glossary/psychosine/) - Due to the enzyme deficiency in Krabbe disease, psychosine accumulates in the nervous system of affected individuals. Psychosine, a galactolipid, is a cytotoxic type of lipid, that has destructive properties affecting the production of myelin.
- [Pathogenic Variant](https://krabbeconnect.org/glossary/pathogenic-variant/) - A variant is anychange in the DNA sequence away from what is considered normal or typical. A change that does not have medical consequences is described as benign, while disease-causing variants are called, pathogenic variants.
- [Palliative Care](https://krabbeconnect.org/glossary/palliative-care/) - Specialized and supportive care for individuals with a serious illness with the goal of offering an improved quality of life, not only for the patient but the family as well.
- [Oligodendrocytes](https://krabbeconnect.org/glossary/oligodendrocytes/) - A type of glial cell found in the central nervous system responsible for the production of myelin.
- [Neurodegenerative Disease](https://krabbeconnect.org/glossary/neurodegenerative-disease/) - This group of diseases is debilitating, progressive and incurable resulting in degeneration and/or nerve cell death.
- [Nervous System](https://krabbeconnect.org/glossary/nervous-system/) - The nervous system of the body is made up of the Central Nervous System (CNS) and the Peripheral Nervous System (PNS).
- [Neuron](https://krabbeconnect.org/glossary/neuron/) - A nerve cell specializing in transmitting nerve impulses.
- [Myelin](https://krabbeconnect.org/glossary/myelin/) - Myelin is an essential material your body uses to surround and protect nerve fibers.
- [Mutations](https://krabbeconnect.org/glossary/mutations/) - This is an older word used to describe a change in a specific gene leading to disease. As not all mutations are bad, the word mutation has been replaced with the term “pathogenic variant” that describes a disease-causing gene.
- [Muscle Atrophy](https://krabbeconnect.org/glossary/muscle-atrophy/) - Descriptive term used to describe when a muscle isn’t working/functioning properly and loses its mass. Often describes as muscle wasting.
- [Metabolic Disease (Disorder)](https://krabbeconnect.org/glossary/metabolic-disease-disorder/) - A group of disorders responsible for abnormal chemical reactions that alter the normal metabolic process. Metabolic diseases are single-gene disorders (Mendelian) inherited in an autosomal recessive pattern.
- [Lysosomal Storage Disease](https://krabbeconnect.org/glossary/lysosomal-storage-disease/) - Lysosomal storage disorders comprise a group of ~50 metabolic disorders that result from a missing, deficient or lack of a specific enzyme.
- [Lipidosis](https://krabbeconnect.org/glossary/lipidosis/) - A lipid storage disorder resulting from harmful amounts of lipids, like the galactolipids, galactoceramide and psychosine, accumulating in cells throughout the body.
- [Lipids](https://krabbeconnect.org/glossary/lipids/) - In Krabbe disease a lipid describes a type of fatty storage material that accumulates in cells.
- [Leukodystrophy](https://krabbeconnect.org/glossary/leukodystrophy/) - The leukodystrophies comprise a group of progressive, genetic disorders mainly affecting the central nervous system (CNS). Most leukodystophies result from a disruption of the growth of the myelin sheath, which provides insulation for nerves.
- [Leukocyte](https://krabbeconnect.org/glossary/leukocyte/) - A type of white blood cell of the immune system that helps the body fight infection.
- [Inherited](https://krabbeconnect.org/glossary/inherited/) - The way genes are passed down from one generation to the next. There are many different types of inheritance patterns. Krabbe disease is inherited in an autosomal recessive pattern of inheritance.
- [HSCT](https://krabbeconnect.org/glossary/hsct/) - Hematopoietic stem cell transplantation (HSCT), a type of transplantation using multipotent hematopoietic stem cells typically derived from bone marrow, peripheral blood, or that from umbilical cord blood. When your own stem cells are used, it’s called autologous, allogeneic when stem cells are used from a donor, and syngeneic when the stem cells are derived from
- [Hypotonia](https://krabbeconnect.org/glossary/hypotonia/) - Synonymous with muscle weakness.
- [Hypertonia](https://krabbeconnect.org/glossary/hypertonia/) - Often described as rigidity and/or spasticity as a result of damage to motor neurons of the central nervous system.
- [HLA Match](https://krabbeconnect.org/glossary/hla-match/) - The success of a bone marrow and cord blood transplants relies on human leukocyte antigen (HLA) marker matching between donors and recipients. HLA are proteins, types of markers, found throughout the body that signal the immune system to identify foreign cells from non-foreign cells. A close match between a donor’s and patient’s HLA markers is paramount
- [Globoid Cell Leukodystrophy](https://krabbeconnect.org/glossary/globoid-cell-leukodystrophy/) - Another name for Krabbe disease, a rare and usually fatal disorder of the nervous system.
- [Glial Cells](https://krabbeconnect.org/glossary/glial-cells/) - A specific type of cell surrounding neurons (a nerve cell) and the most abundant cell type found in the central nervous system (CNS). Glial cells provide the insulation and support for neurons.There are many types of glial cells including oligodendrocytes, astrocytes, ependymal cells, Schwann cells, microglia, and satellite cells.
- [Genetic Mutations](https://krabbeconnect.org/glossary/genetic-mutations/) - See Mutations
- [Gene Therapy](https://krabbeconnect.org/glossary/gene-therapy/) - A type of therapy that offers hope and promise for a cure for many genetic disorders. A working copy of the gene replaces the non-working copy of the gene. Gene therapy is at the forefront of many treatments for genetic disorders like Krabbe disease.
- [Globoid Cells](https://krabbeconnect.org/glossary/globoid-cells/) - The abnormal cells found in the brain of patients with Krabbe disease have a characteristic pathology known as globoids cells, which are large cells usually with more than one nucleus.
- [Genetic Disease](https://krabbeconnect.org/glossary/genetic-disease/) - A disease or disorder caused by a pathogenic variant in a gene, most often present from birth. There are ~8000 genetic diseases identified to date. Other types of diseases can be caused by chromosomal changes and environmental influences.
- [Genes](https://krabbeconnect.org/glossary/genes/) - The body is made of ~20,500 genes, each of which has a specific function within the body. Our genes are what makes each one of us unique- from our green eyes to our curly hair.
- [Galactosylceramide](https://krabbeconnect.org/glossary/galactosylceramide/) - A galactolipid that is an important component of the normal turnover of myelin in nerve fibers. When there is an enzyme deficiency, excess galactosylceramide and psychosine (both galactolipids) store in certain cells- forming the globoid cells seen in Krabbe disease and the consequential damage found in myelin-forming cells. This damage leads to demyelination in the
- [Galactoceramidase](https://krabbeconnect.org/glossary/galactoceramidase/) - Another name for galactocerebrosidase.
- [Enzyme](https://krabbeconnect.org/glossary/enzyme/) - A protein needed by the body to break down certain substances by causing a biochemical reaction within the cells. In Krabbe disease, galactocerebrosidase is the deficient enzyme. Words that end in ‘ase’ are usually identified as an enzyme.
- [Demyelination](https://krabbeconnect.org/glossary/demyelination/) - A process whereby the body breaks down the protective coating surrounding nerves.
- [DNA](https://krabbeconnect.org/glossary/dna/) - An abbreviation for deoxyribonucleic acid (DNA). DNA is the genetic blueprint or the instruction manual for the human body. While we appear so unique, it is amazing to note that two unrelated individuals are more than 99.9% similar. Our DNA is composed of a double-helical, spiral like ladder and the length of our DNA is
- [Clinical Trial](https://krabbeconnect.org/glossary/clinical-trial/) - A clinical trial is research designed to understand the safety and efficacy of a drug, biologic or device. There are 4 phases to most clinical trials from Phase 1 that seeks to answer safety concerns in a healthy population of volunteers to Phase 2 trials aimed at understanding the safety of the product in a
- [Central Nervous System](https://krabbeconnect.org/glossary/central-nervous-system/) - The central nervous system (CNS) is made up of the brain and the spinal cord.
- [Cord Blood Transplant](https://krabbeconnect.org/glossary/cord-blood-transplant/) - See Umbilical Cord Blood Transplant (UCBT).
- [Bone Marrow Transplant](https://krabbeconnect.org/glossary/bone-marrow-transplant/) - A medical procedure whereby diseased bone marrow is replaced with healthy bone marrow. Often times, the patients’ bone marrow will be destroyed by high doses of certain chemotherapies and/or radiation therapy.
- [Autosomal Recessive Inheritance](https://krabbeconnect.org/glossary/autosomal-recessive-inheritance/) - A pattern of inheritance whereby both parents must be a carrier for the disease in order to have a child with the disorder. Even when both parents are carriers for the disorder, there is a 1 in 4 chance the child will inherit the disorder, a 1: 2 chance the child will also be a carrier
- [AAV10 Vector](https://krabbeconnect.org/glossary/aav10-vector/) - AAV stands for Adeno-Associated Virus and holds promise for the treatment and/or cure of many genetic disorders that have a neurodegenerative component. As the name implies, there are many types of AAV viruses and the AAV10 vector has shown promise in crossing the blood-brain barrier- a key obstacle to the delivery of gene therapy to

## Categories

- [Medical Journals](https://krabbeconnect.org/category/resources/medical-journals/)
- [Patient Journey and Resources Maps](https://krabbeconnect.org/category/resources/patient-journey-and-resources-maps/)
- [Grandparent of Krabbe Hero](https://krabbeconnect.org/category/grandparent-of-krabbe-hero/)
- [Resources](https://krabbeconnect.org/category/resources/)
- [Videos](https://krabbeconnect.org/category/resources/videos/)
- [Press Release](https://krabbeconnect.org/category/press-release/)

## Categories

- [Medical Journals](https://krabbeconnect.org/library-category/medical-journals/)
- [Videos](https://krabbeconnect.org/library-category/videos/)
- [Patient Journey and Resources Maps](https://krabbeconnect.org/library-category/patient-journey-and-resources-maps/)

## Status

- [Currently Screening](https://krabbeconnect.org/state-status/currently-screening/)
- [RUSP Alignment Policy In Place](https://krabbeconnect.org/state-status/bill-filed/)
- [Ongoing Advocacy Efforts to Advance NBS](https://krabbeconnect.org/state-status/bill-passed-not-screening/)
- [Implementation in Progress](https://krabbeconnect.org/state-status/implementation-in-progress/)