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

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mRNA Treatment Rescues Niemann–Pick Disease Type C1 in Patient Fibroblasts

Understanding the phenotypic variability in Niemann-Pick disease type C (NPC): a need for precision medicine

Diagnostic workup and management of patients with suspected Niemann-Pick type C disease - Apostolos Papandreou, Paul Gissen, 2016

Adult Onset Niemann-Pick type C (NPC) – NNPDF

Fig 1., Subcortical Volumetric Reductions in Adult Niemann-Pick Disease Type C: A Cross-Sectional Study

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

Niemann-Pick disease type C, Orphanet Journal of Rare Diseases

Recommendations on the diagnosis and management of Niemann-Pick disease type C - ScienceDirect

Niemann-Pick Type C Disease

FDA needs to be more flexible assessing rare disease treatments - STAT

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

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