Treating Niemann-Pick Disease Type C
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Treatment outcomes following continuous miglustat therapy in patients with Niemann-Pick disease Type C: a final report of the NPC Registry, Orphanet Journal of Rare Diseases
Niemann-Pick disease type C
Cyclodextrin News
Why are people fighting over a promising treatment for a fatal childhood disease?, Science
Niemann–Pick Disease Type C
Summer 2022 Newsletter – NNPDF
Parents of kids with Niemann Pick C advocate for adrabetadex
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
Consensus clinical management guidelines for Niemann-Pick disease type C, Orphanet Journal of Rare Diseases
Main therapies and point of action in Niemann type C disease.
Potential treatment for Niemann-Pick type C, a rare neurodegenerative disease
New therapies for Niemann-Pick Disease Type C treatment by DelveInsight Business Research LLP., DelveInsight Business Research LLP posted on the topic
Elevation of plasma lysosphingomyelin-509 and urinary bile acid metabolite in Niemann-Pick disease type C-affected individuals - Top Articles selected by the Editor - Molecular Genetics and Metabolism Reports - Journal - Elsevier
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