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

A Practical Chromogenic Procedure for the Detection of Homozygotes and Heterozygous Carriers of Niemann-Pick Disease

Niemann-Pick Disease Concise Medical Knowledge

Valley family fights for drug to save sick child

NPC1 enables cholesterol mobilization during long‐term potentiation that can be restored in Niemann–Pick disease type C by CYP46A1 activation

Intrathecal cyclodextrin in the treatment of Niemann-Pick disease type C

Enhanced Delivery and Effects of Acid Sphingomyelinase by ICAM-1-Targeted Nanocarriers in Type B Niemann-Pick Disease Mice - ScienceDirect

5 Most Promising Companies offering hope to patients with Niemann-Pick Type C

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

Treatment outcomes following continuous miglustat therapy in patients with Niemann-Pick disease Type C: a final report of the NPC Registry, Orphanet Journal of Rare Diseases

Niemann-Pick disease - News, Articles etc. - European Pharmaceutical Review

Parents of twins with Niemann-Pick find a treatment

TRIALS: A Fight for Children with Niemann-Pick Type C, a Fatal Genetic Disease

Niemann-Pick Disease, A Pipeline Analysis Report 2018, Technavio

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