Listen and Learn Webinars

Bone marrow transplants are utilized in patients diagnosed with Globoid Cell LeukodystrophyAnother name for Krabbe disease, a rare and usually fatal disorder of the nervous system. (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 non-malignant disorders such as Krabbe disease. Dr. Paul Szabolcs will present his recent publication discussing the use of RIC in patients with Krabbe disease. Dr. Paul Szabolcs is the Division Director at the Blood and Marrow Transplantation and Cellular Therapies at the University of Pittsburgh.

Join us for a special caregiver’s webinar. From a fairytale family to losing three brothers, mental health advocate Cristol Barrett O’Loughlin draws on personal tragedy to ease the suffering hearts of others. Her inspiring stories of courageous #RareMothers, reveal the secrets to sustainable self-care and wellness. With the onslaught of COVID-19, we are collectively living with the realities and stressors of home/work life balance, homeschooling, medical uncertainty. There is a purpose to our pain. With guided meditations and her signature infectious enthusiasm, Cristol gently nudges us all to move beyond “Why me?” into “What can I do to help others and to help myself?”

Are you asking yourself….how can I make more of an impact in Krabbe disease or I wonder if I should start my own nonprofit foundation to advance the work in Krabbe disease? If you are contemplating these very questions, we strongly encourage you to join us for a 1-hour webinar to learn more about the Partner with Us program. This unique program allows you to name a fund, maintain ownership of the fund, and select projects that resonate with you to help #curekrabbe.

There’s a lot of information to evaluate when clinical trial becomes available. For example, who is able to enroll, what does exclusion and inclusion criteria mean, how does a clinical trial work and so much more. This 1-hour webinar is crafted to help you easily navigate a clinical trial and answer some questions about the new Krabbe disease clinicals trials offered by Forge Biologics, Passage Bio, and Polaryx.

Join us for a 1-hour discussion with The National Organization for Rare Disorders and KrabbeConnect on KrabbeCURES, a new tool to  help fill gaps in research on study globoid cell leukodystrophyThe 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 sh (Krabbe disease). KrabbeCURES is a versatile online system that securely collects and stores data for medical research.  It is a dynamic participant-driven resource that can empower and unite the globoid cell leukodystrophy community through shared knowledge.  We want all Krabbe disease stakeholders (patients, caregivers, clinicians, researchers, industry, etc.) to understand this new tool and how it will help us #curekrabbe.

A 1-hour educational discussion with Dr. Joanne Kurtzberg on the history of transplant in Krabbe disease, lessons learned since utilizing transplants to treat Krabbe disease, and some useful insights on new therapies in the pipeline.  Dr. Joanne Kurtzberg is an internationally renowned expert in pediatric hematologyoncology, pediatric blood and marrow transplantation, umbilical cord blood banking and transplantation, and novel applications of cord blood in the emerging fields of cellular therapies and regenerative medicine. Since 1990 she has served as the Director of the Pediatric Blood and Marrow Transplant (PBMT) Program at Duke University Medical Center. The PBMT program has transplanted over 60 patients affected by Krabbe Disease. Dr. Kurtzberg current research is focused on finding ways to use cord blood cells to help children with Krabbe disease and other leukodystrophies.

A 45-minute presentation with guest speaker, Debbie Drell from the National Organization for Rare Disorders (NORD).  Debbie, along with Co-Founders, Stacy Pike-Langenfeld and Anne Rugari, highlight all the elements of a Patient Focused Drug Development meeting and why every stakeholder (patients, caregivers, drug developers, clinicians, researchers, etc.) need to come together to inform the FDA about Krabbe disease, the impact KD has on a patient’s life, current treatments, and the need for new therapies. 

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 DiseaseLysosomal storage disorders comprise a group of ~50 metabolic disorders that result from a missing, deficient or lack of a specific enzyme. Center, and member of the Emory Biomedical IRB Committee.  Dawn is a long-time advocate of patient empowerment and strongly urges rare disease patients and/or caregivers to organize a care team to ensure the exchange of information amongst different medical care providers remains fluid and consistent.