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

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NIH clinical trial begins for treatment of rare, fatal neurological disorder

Experimental treatment for Niemann-Pick disease type C1 appears safe, effective

When Drug Development for Rare Disease Hit Setback, Parents Were Stung - WSJ

February 2023 Newsletter – NNPDF

Niemann–Pick type C disease as proof‐of‐concept for intelligent biomarker panel selection in neurometabolic disorders - Papandreou - 2022 - Developmental Medicine & Child Neurology - Wiley Online Library

Niemann–Pick type C disease: cellular pathology and pharmacotherapy - Wheeler - 2020 - Journal of Neurochemistry - Wiley Online Library

Niemann-Pick Type C Disease

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

PDF) Annual severity increment score as a tool for stratifying patients with Niemann-Pick disease type C and for recruitment to clinical trials

Miglustat in Niemann-Pick disease type C patients: a review, Orphanet Journal of Rare Diseases

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

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

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

Patient with Niemann-Pick disease type C: over 20 years' follow-up