Experimental Treatment for Niemann-Pick Disease Type C1 Appears Safe, EffectiveAn experimental drug appears to slow the progression of Niemann-Pick disease type C1 — a fatal neurological disease that primarily affects children and adolescents, causing a progressive decline in neurological and cognitive functions — according to results of a clinical study led by researchers at the National Institutes of Health. The drug, 2-hydroxypropyl-beta-cyclodextrin (VTS-270), is being tested under a cooperative research and development agreement, or CRADA, between NIH and Sucampo Pharmaceuticals, Inc.
