Thursday - August 17, 2017
Experimental Treatment for Niemann-Pick Disease Type C1 Appears Safe, Effective
August 11, 2017
BETHESDA, Md., Aug. 11 -- The National Institutes of Health's National Institute on Deafness and Other Communication Disorders issued the following news:

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 an . . .

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