Niemann-Pick B - RS - 5th infusion.

PDF) Chronic visceral acid sphingomyelinase deficiency (Niemann-Pick disease type B) in 16 Polish patients: Long-term follow-up

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

Natural history and management of liver dysfunction in lysosomal storage disorders

Enhanced Delivery and Effects of Acid Sphingomyelinase by ICAM-1-Targeted Nanocarriers in Type B Niemann-Pick Disease Mice - ScienceDirect

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)

Biomolecules, Free Full-Text

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

Interstitial lung disease in lysosomal storage disorders

Potential treatment for Niemann-Pick type C, a rare neurodegenerative disease

PDF) Reducing GBA2 Activity Ameliorates Neuropathology in Niemann-Pick Type C Mice

Splenomegaly, hypersplenism, and hereditary disorders with splenomegaly

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