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

Valley baby showing great progress with experimental Niemann-Pick Type C disease drug treatments

Neurological manifestations of lysosomal disorders and emerging therapies targeting the CNS - The Lancet Child & Adolescent Health

Familial Hypercholesterolemia: From Clinical Suspicion to Novel Treatments

Health Tip on New Treatment that Shows Promise in Niemann-Pick Type C Disorder - Health Tips

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

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

Lithium ameliorates Niemann-Pick C1 disease phenotypes by impeding STING/SREBP2 activation - ScienceDirect

Orphazyme finds reduced Niemann-Pick disease progression in trial

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

Dermatologic Manifestations of Niemann-Pick Disease: Background, Pathophysiology, Etiology

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

TPC2 rescues lysosomal storage in mucolipidosis type IV, Niemann–Pick type C1, and Batten disease

Experimental treatment for Niemann-Pick disease type C1 appears safe, effective