Experimental treatment for Niemann-Pick disease appears safe

Por um escritor misterioso

Last updated 20 março 2025

An experimental drug has appeared to slow the progression of Niemann-Pick disease the drug 2-hydroxypropyl-beta-cyclodextrin is being tested

Gene therapy shows promise for treating Niemann-Pick disease type C1

Current advancements in therapy for Niemann-Pick disease: progress and pitfalls: Expert Opinion on Pharmacotherapy: Vol 24, No 11

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

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

IJMS, Free Full-Text

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

Emerging Startups Take Aim at Lysosomal Storage Disorders

Molecular Mind Games: The Medicinal Action of Cyclodextrins in Neurodegenerative Diseases

Recomendado para você