Niemann-Pick B - RS - 5th infusion.

Interstitial lung disease in lysosomal storage disorders

IJMS, Free Full-Text

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

JCM, Free Full-Text

Frontiers Potential Disease-Modifying Effects of Lithium Carbonate in Niemann-Pick Disease, Type C1

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

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

Investigating the Mechanism of Cyclodextrins in the Treatment of Niemann‐ Pick Disease Type C Using Crosslinked 2‐Hydroxypropyl‐β‐cyclodextrin - Carradori - 2020 - Small - Wiley Online Library

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

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)

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

Interstitial lung disease in lysosomal storage disorders