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 new treatment can delay effects of Neimann-Pick disease in children

Miglustat in Niemann-Pick disease type C patients: a review, Orphanet Journal of Rare Diseases

IJMS, Free Full-Text

Families plead with FDA for drug to treat Niemann-Pick Disease Type C

Valley boy battling rare disease getting stronger

Niemann–Pick type C disease: cellular pathology and pharmacotherapy - Wheeler - 2020 - Journal of Neurochemistry - Wiley Online Library

Niemann Pick Disease Type C - Symptoms, Causes, Treatment

Adenosine A2A receptor stimulation restores cell functions and differentiation in Niemann-Pick type C-like oligodendrocytes

Frontiers c-Abl Activation Linked to Autophagy-Lysosomal Dysfunction Contributes to Neurological Impairment in Niemann-Pick Type A Disease

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

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