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

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

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

Frontiers Potential Disease-Modifying Effects of Lithium Carbonate in Niemann-Pick Disease, Type C1

Niemann-Pick Disease, Type a disease: Malacards - Research Articles, Drugs, Genes, Clinical Trials

INPDA International Niemann-Pick Disease Alliance

Correlation of age of onset and clinical severity in Niemann–Pick disease type C1 with lysosomal abnormalities and gene expression

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

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

Emerging Startups Take Aim at Lysosomal Storage Disorders

Full article: Current advancements in therapy for Niemann-Pick disease: progress and pitfalls

Therapeutic intervention for Niemann–Pick type C disease must target