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 your idea/s at firstname.lastname@example.org
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Webinars Available for Download
Brain and Tissue Banking: Why It's Important and Is It Right For Me?
Wednesday, January 19, 2022 4:00 PM EST / 3:00PM CST
The importance of human organ and tissue donation for research can be a difficult decision to make. For others, this donation may offer a sense of purpose and comfort, knowing that the quest for knowledge and medical research will carry on for Krabbe Disease. Brain and Tissue Banking for Krabbe and other rare diseases allow researchers to better understand the changes taking place in the brain and other tissues in the body. This type of research can lead to new breakthroughs and testing of novel ideas that eventually translates into a potential treatment and a possible cure. This webinar hosts four families who have donated their child’s brain and tissues to the NDRD Brain and Tissue Bank. You will hear their stories on why they chose to donate, the process, and their personal comments on why it was an important choice for their family. You will also hear from Dr. Julia Kofler, who will explain the importance of banking from a researcher’s perspective. She will present information on projects that have come from the Krabbe family’s donations. Dr. Kofler is the Co-director and Neuropathology Core Leader at the Alzheimer’s Disease Research Center, University of Pittsburgh, where the Krabbe brain and tissues are stored.
Rationale for Reduced-Intensity Conditioning (RIC) Transplantation in Krabbe Disease
Tuesday, November 9th at 4:00 pm EST/3:00 pm CST
Bone 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 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.
Caring for the Caregivers: Sensory Self-Care in Micro-Moments a Day
Original Record Date: May 3rd, 2021
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?”
Partner with Us Program: How to Set Your Own Fund
Original Record Date: Monday, April 12th, 2021
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.
Clinical Trials 101: Key Things to Know About Studies in Krabbe disease
Original Record Date: Sunday, March 21st, 2021
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.
Advancing Research Through the Krabbe Community United Research and Engagement Study (KrabbeCURES)
Original Record Date: December 15, 2020
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 leukodystrophy (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.
Krabbe Disease: Transplants and Arising New Therapies
Original Air Date: June 29th, 2020
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 hematology\oncology, 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.
Be Heard By the FDA: Learn More About the Patient Focused Drug Development Meeting for Krabbe Disease
Original Air Date: June 16th, 2020
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.
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 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.
COVID-19 Insights for Krabbe Families
Original Air Date: April 3rd, 2020
A 1-hour presentation with Dr. Maria Escolar, a tenured Professor of Pediatrics and Neurodevelopmental Disabilities at the University of Pittsburgh. Dr. Escolar comes to KrabbeConnect with more than 20 years of experience as a clinician treating patients with Krabbe disease and other rare neurological conditions. Dr. Escolar provides an overview of COVID-19 and concerns related to patients already compromised by Krabbe disease.