Niemann-Pick B - RS - 5th infusion.

Natural history and management of liver dysfunction in lysosomal storage disorders

Advances in therapies for neurological lysosomal storage disorders - Ellison - 2023 - Journal of Inherited Metabolic Disease - Wiley Online Library

Long-term administration of intravenous Trappsol® Cyclo™ (HP-β-CD) results in clinical benefits and stabilization or slowing of disease progression in patients with Niemann-Pick disease type C1: Results of an international 48-week Phase I/II

Niemann-Pick C1 expression is not regulated by the amount of cholesterol flowing through cells in the mouse - ScienceDirect

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

Frontiers Expert opinion on patient journey, diagnosis and clinical monitoring in acid sphingomyelinase deficiency in Turkey: a pediatric metabolic disease specialist's perspective

Natural history and management of liver dysfunction in lysosomal storage disorders

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

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

PDF) Novel first-dose adverse drug reactions during a phase I trial of olipudase alfa (recombinant human acid sphingomyelinase) in adults with Niemann–Pick disease type B (acid sphingomyelinase deficiency)