Experimental treatment for Niemann-Pick disease appears safe

An experimental drug has appeared to slow the progression of Niemann-Pick disease the drug 2-hydroxypropyl-beta-cyclodextrin is being tested

PDF) Recent Advances in the Diagnosis and Treatment of Niemann-Pick Disease Type C in Children: A Guide to Early Diagnosis for the General Pediatrician

INPDA International Niemann-Pick Disease Alliance

Deaf or Death? In Drug Trial, Parents Weigh Life vs. Hearing Loss - WSJ

Emerging Startups Take Aim at Lysosomal Storage Disorders

Ataxia–telangiectasia - Wikipedia

Niemann-Pick disease Information

Niemann-Pick disease A or B in four pediatric patients and SMPD1 mutation carrier frequency in the Mexican population

Psychiatric and Cognitive Symptoms Associated with Niemann-Pick Type C Disease: Neurobiology and Management

Experimental Treatment for Niemann-Pick Disease Type C1 Appears Safe, Effective