Experimental treatment for Niemann-Pick disease appears safe
Por um escritor misterioso
Last updated 16 abril 2025

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 Information

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

Emerging Startups Take Aim at Lysosomal Storage Disorders

Experimental treatment for Niemann-Pick disease appears safe

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

Improved systemic AAV gene therapy with a neurotrophic capsid in Niemann–Pick disease type C1 mice

Intrathecal cyclodextrin in the treatment of Niemann-Pick disease type C

Acid Sphingomyelinase Deficiency disease: Malacards - Research Articles, Drugs, Genes, Clinical Trials

Niemann-Pick disease type C: Video & Anatomy

Tolerance of chronic HDACi treatment for neurological, visceral and lung Niemann-Pick Type C disease in mice
Current advancements in therapy for Niemann-Pick disease: progress and pitfalls: Expert Opinion on Pharmacotherapy: Vol 24, No 11

Frontiers Case Report: Be Aware of “New” Features of Niemann–Pick Disease: Insights From Two Pediatric Cases
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