Miglustat for treatment of Niemann-Pick C disease: a randomised

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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

List Niemann-Pick as rare disease, parents of young patients urge

Niemann Pick disease

Experimental treatment for Niemann-Pick disease appears safe

Niemann-Pick disease type C. The diagram represents subtypes and

Recommendations on the diagnosis and management of Niemann-Pick

Treatment outcomes following continuous miglustat therapy in

Hepatocellular carcinoma as a complication of Niemann‐Pick disease

Niemann-Pick Disease, Types C1 (D)