Rare Diseases Research: Clinical Trial for Niemann-Pick Type C
Science Talk
English - June 03, 2014 18:57 - 10 minutes - 85.1 MB Video - ★★★★ - 8 ratingsCourses Education Science yeshiva university medical school Homepage Download Apple Podcasts Google Podcasts Overcast Castro Pocket Casts RSS feed
https://www.einstein.yu.edu - Determined to find a treatment for children with the degenerative brain disease Niemann-Pick Type C, Steven Walkley, D.V.M., Ph.D., turned a serendipitous laboratory discovery into a successful national research collaboration with other academic institutions and the National Center for Advancing Translational Sciences' program for rare diseases (Therapeutics for Rare and Neglected Diseases). These efforts led to an NIH Phase 1 clinical trial testing cyclodextrin
as a therapy for children with this disease. Dr. Walkley is a professor in the Dominick P. Purpura Department of Neuroscience and director of the Rose F. Kennedy Intellectual and Developmental Disabilities Research Center at Albert Einstein College of Medicine.