Experimental treatment for Niemann-Pick disease type C1 appears safe, effective
NIH-led clinical trial suggests that drug slows progression of rare neurological disease.
An experimental drug appears to slow the progression of Niemann-Pick disease type C1 (NPC1), a fatal neurological disease, according to results of a clinical study led by researchers at the National Institutes of Health. The study appears in The Lancet.
NPC1 is a rare genetic disorder that primarily affects children and adolescents, causing a progressive decline in neurological and cognitive functions. The U.S. Food and Drug Administration has not approved any treatments for the condition.
This page was last updated on Friday, January 21, 2022