2012•Pediatric NeurologyRequires access

Early Miglustat Therapy in Infantile Niemann-Pick Disease Type C

Maja Di Rocco, Andrea Dardis, Annalisa Madeo, Rita Maria Elisa Barone, Agata Fiumara

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Key concepts: Niemann–Pick disease, type C, Substrate reduction therapy, Medicine, Pediatrics, Niemann–Pick disease, Asymptomatic, Lysosomal storage disease, Disease

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Early Miglustat Therapy in Infantile Niemann-Pick Disease Type C — Research Paper | ScholarLens