Read about the detailed efforts of the Marellas and other NP-C families to bring the latest and most promising therapy for NP-C, Cyclodextrin, into an FDA approved clinical trial in an amazing, online experience! Trials: A Desperate Fight to Save Kids & Change Science. For six years, Wall Street Journal reporter Amy Dockser Marcus followed …
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Niemann-Pick Type C News
DART and SOAR-NPC Researcher Dr. Charles Vite in the University of Pennsylvania News!
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 …