Educational Videos & Webinars

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, validation, and connection during a difficult season.

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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 Krabbe Disease, this webinar offers critical updates and data-driven insights into these groundbreaking studies.

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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 be like after a Krabbe disease diagnosis.

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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 medicine at Albert Einstein Medical Center in Philadelphia, PA. Dr. Bascou, a physician resident in Neurology at Johns Hopkins University states, "It is incredible to see the leaps in our understanding of Krabbe disease over the past decade and the new treatments that are becoming available. Our team is extremely grateful to RFRF for providing the resources needed to help conduct a study that we hope will demonstrate how big of an impact NBS and disease-modifying therapies have on the lives of patients and their families affected by Krabbe."

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 received transplants and those who haven't, the research aims to shed light on potential differences in outcomes, informing future treatment strategies, patient care, and most importantly, the importance of early intervention.

We invite you to take 15 minutes to learn more! We encourage everyone to take the initial survey whether you qualify or not! This allows us to gather insights on the current Krabbe disease demographics. Those who qualify for the study will be asked to participate in a 60-90 minute interview. Start your survey here- https://www.engagehealth.com/survey/LoginCode.aspx?SurveyID=8lKJ6l53

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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 globoid cell leukodystrophyAnother name for Krabbe disease, a rare and usually fatal disorder of the nervous system.. Krabbe disease is a severe neurodegenerative and rapidly progressing condition requiring immediate treatment for the most severe forms. A delayed diagnosis, especially in the most severe forms, equates to palliative and supportive care as the only means of treatment until premature death.

We invite you to learn more about this disease and all the heroes impacted by this neurological disease at https://krabbeconnect.org/

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This video was first debuted at the 2021 KrabbeConnect, "A Million Dreams," gala. Dr. Allison Bradbury has evaluated disease mechanisms and developed gene therapyA 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 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 safer and more efficient viral vehicles to deliver therapeutic transgenes in a single injection, and gene therapy is now a favorable therapeutic intervention for monogenic diseases.

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