Niemann-Pick B - RS - 5th infusion.
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Advances in therapies for neurological lysosomal storage disorders - Ellison - 2023 - Journal of Inherited Metabolic Disease - Wiley Online Library
Neurological manifestations of lysosomal disorders and emerging therapies targeting the CNS - The Lancet Child & Adolescent Health
Interstitial lung disease in lysosomal storage disorders
PDF) Reducing GBA2 Activity Ameliorates Neuropathology in Niemann-Pick Type C Mice
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
AAV-PHP.B Administration Results in a Differential Pattern of CNS Biodistribution in Non-human Primates Compared with Mice: Molecular Therapy
TAVI in Patient Suffering from Niemann–Pick Disease (Acid Sphingomyelinase Deficiency) with Concomitant Situs Inversus and Dextrocardia
Sphingomyelin-induced inhibition of the plasma membrane calcium ATPase causes neurodegeneration in type A Niemann–Pick disease
PDF) A Prospective, Cross-sectional Survey Study of the Natural History of Niemann-Pick Disease Type B
Lysosomal enzyme replacement therapies: Historical development, clinical outcomes, and future perspectives. - Abstract - Europe PMC
Distribution of acid sphingomyelinase in rodent and non-human primate brain after intracerebroventricular infusion - ScienceDirect
Frontiers Potential Disease-Modifying Effects of Lithium Carbonate in Niemann-Pick Disease, Type C1
Natural history and management of liver dysfunction in lysosomal storage disorders
Frontiers Expert opinion on patient journey, diagnosis and clinical monitoring in acid sphingomyelinase deficiency in Turkey: a pediatric metabolic disease specialist's perspective
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