Niemann-Pick B - RS - 5th infusion.

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Frontiers Pre-clinical Mouse Models of Neurodegenerative Lysosomal Storage Diseases

Niemann-Pick disease type C

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

Natural history and management of liver dysfunction in lysosomal storage disorders

PDF) Consensus clinical management guidelines for Acid Sphingomyelinase Deficiency (Niemann-Pick disease types A, B and A/B)

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

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

Experimental treatment for Niemann-Pick disease type C1 appears safe, effective

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