Niemann Pick Disease Type C - Symptoms, Causes, Treatment
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Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study - The Lancet Neurology
At a glance: the largest Niemann-Pick type C1 cohort with 602 patients diagnosed over 15 years
Niemann-Pick disease type C. The diagram represents subtypes and onset
Niemann–Pick disease - Wikipedia
From genes to hope
Oxidative Stress: A Pathogenic Mechanism for Niemann-Pick Type C Disease
Niemann-Pick disease type C symptomatology: an expert-based clinical description, Orphanet Journal of Rare Diseases
What is Niemann Pick Type C - Race for Adam
Miglustat in Niemann-Pick disease type C patients: a review, Orphanet Journal of Rare Diseases
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