Treating Niemann-Pick Disease Type C
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Full article: New therapies in the management of Niemann-Pick type C disease: clinical utility of miglustat
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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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Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study - The Lancet Neurology
Niemann-Pick Disease, Types C1 (D)
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