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- www.engagehealth.com/survey/LoginCode.aspx?SurveyID=8lKJ6l53