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

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At a glance: the largest Niemann-Pick type C1 cohort with 602 patients diagnosed over 15 years

Potential treatment for Niemann-Pick type C, a rare neurodegenerative disease

Niemann-Pick disease: Causes, symptoms, treatment, and more

Niemann-Pick Type C Disease

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

Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study - The Lancet Neurology

Niemann-Pick Type C News Dana's Angels Research Trust (DART)

Immune dysfunction in Niemann‐Pick disease type C - Platt - 2016 - Journal of Neurochemistry - Wiley Online Library

Australian Research Updates Australian NPC Disease Foundation Inc

Validation of the 5-domain Niemann-Pick type C Clinical Severity Scale

PDF) Consensus clinical management guidelines for Niemann-Pick disease type C