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Rare Diseases Research: Clinical Trial for Niemann-Pick Type C

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

5 Most Promising Companies offering hope to patients with Niemann-Pick Type C

Niemann–Pick Disease Type C

Presentations of Niemann-Pick Type C disease

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Niemann-Pick Disease Type C Archives - The Children's Inn at NIH

Mitochondrial G8292A and C8794T mutations in patients with Niemann‑Pick disease type C Corrigendum in /10.3892/br.2018.1156

The pathogenesis of Niemann–Pick type C disease: a role for autophagy?, Expert Reviews in Molecular Medicine