Niemann-Pick B - RS - 5th infusion.

JCM, Free Full-Text

IJMS, Free Full-Text

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

Niemann-Pick Disease

Autumn 2009 - the Niemann-Pick Disease Group (UK)

Niemann-Pick disease type C

Natural history and management of liver dysfunction in lysosomal storage disorders

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

Niemann-Pick disease type C

Lysosomal storage diseases: Diagnostic confirmation and management of presymptomatic individuals

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