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

Why are people fighting over a promising treatment for a fatal childhood disease?, Science

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

Parents of twins with Niemann-Pick find a treatment

Niemann-Pick disease type C – topic of research paper in Biological sciences. Download scholarly article PDF and read for free on CyberLeninka open science hub.

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

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

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

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

Niemann-Pick Disease Type C (NP-C) Gene Therapy Activation Meeting (GAM) - International Niemann-Pick Disease Alliance (INPDA)

IJMS, Free Full-Text