Niemann-Pick B - RS - 5th infusion.

Lysosomal storage diseases: Diagnostic confirmation and management of presymptomatic individuals

Biomolecules, Free Full-Text

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

Repurposing the Ebola and Marburg Virus Inhibitors Tilorone, Quinacrine, and Pyronaridine: In Vitro Activity against SARS-CoV-2 and Potential Mechanisms

Familial Hypercholesterolemia: From Clinical Suspicion to Novel Treatments

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

Lysosomal storage disorders: from biology to the clinic with reference to India - ScienceDirect

Natural history and management of liver dysfunction in lysosomal storage disorders

Olipudase alfa enzyme replacement therapy for acid sphingomyelinase deficiency (ASMD): sustained improvements in clinical outcomes after 6.5 years of treatment in adults, Orphanet Journal of Rare Diseases

Sphingomyelin-induced inhibition of the plasma membrane calcium ATPase causes neurodegeneration in type A Niemann–Pick disease

Niemann–Pick disease - Wikipedia

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

PDF) Changes in PCSK 9 and Apolipoprotein B100 in Niemann-Pick Disease After Enzyme Replacement Therapy with Olipudase Alfa

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