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Rare Diseases Research: Clinical Trial for Niemann-Pick Type C

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, part of the NIH. 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 Einstein. (April 29, 2014)
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