Niemann-Pick B - RS - 5th infusion.

Current Clinical Applications of In Vivo Gene Therapy with AAVs: Molecular Therapy

PDF) Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1-2 trial

Expanded access with intravenous hydroxypropyl-β-cyclodextrin to treat children and young adults with Niemann-Pick disease type C1: a case report analysis, Orphanet Journal of Rare Diseases

Lysosomal Storage Disease disease: Malacards - Research Articles, Drugs, Genes, Clinical Trials

Familial Hypercholesterolemia: From Clinical Suspicion to Novel Treatments

Interstitial lung disease in lysosomal storage disorders

PDF) Defects in sarcolemma repair and skeletal muscle function after injury in a mouse model of Niemann-Pick type A/B disease

Pediatric hepatocellular carcinoma associated with Niemann–Pick disease type C: Case report and literature review - Hwang - 2023 - JIMD Reports - Wiley Online Library

Fluorinated Chaperone−β-Cyclodextrin Formulations for β-Glucocerebrosidase Activity Enhancement in Neuronopathic Gaucher Disease