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

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

Promoter considerations in the design of lentiviral vectors for use in treating lysosomal storage diseases: Molecular Therapy - Methods & Clinical Development

Lysosomal storage diseases: Diagnostic confirmation and management of presymptomatic individuals - Genetics in Medicine

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

Interstitial lung disease in lysosomal storage disorders

British Journal of Haematology

Interstitial lung disease in lysosomal storage disorders

IJMS, Free Full-Text

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

Sphingomyelin-induced inhibition of the plasma membrane calcium ATPase causes neurodegeneration in type A Niemann–Pick disease

Lysosomal storage disorders: from biology to the clinic with reference to India - ScienceDirect

IJMS, Free Full-Text

Frontiers Pre-clinical Mouse Models of Neurodegenerative Lysosomal Storage Diseases

PDF) Intestinal Absorption of -Tocotrienol Is Mediated by Niemann-Pick C1-Like 1: In Situ Rat Intestinal Perfusion Studies