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.
Niemann-Pick disease type C1, a lipid storage disorder, as seen in a mouse cerebellum.
This page was last updated on Friday, January 21, 2022