Penn Vet Research on Niemann-Pick Disease Paves Way for Human Clinical Trial February 8, 2013 PHILADELPHIA — Niemann-Pick Type C disease is a rare and incurable neurological disorder that affects 500 children worldwide. Presently, there are no therapies approved by the U.S. Food and Drug Administration to treat NPC. But Charles Vite, an associate professor …
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Cyclodextrin Drug Trial Update
Cyclodextrin Drug Trial Update November 14, 2012 From Dr. Forbes Porter, Clinical Director of the Division of Intramural Research at the National Institute of Child Health and Human Development, National Institutes of Health in Bethesda, Maryland. The Therapies for Rare and Neglected Diseases (TRND) team is pleased to announce that the Investigational New Drug (IND) …