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

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

Tolerance of chronic HDACi treatment for neurological, visceral and lung Niemann-Pick Type C disease in mice

Parents of twins with Niemann-Pick find a treatment

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

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

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

Valley family fights for drug to save sick child

Therapeutic intervention for Niemann–Pick type C disease must target

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

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

Juvenile CLN3 disease is a lysosomal cholesterol storage disorder: similarities with Niemann-Pick type C disease - eBioMedicine