Rare Diseases Research: Clinical Trial for Niemann-Pick Type C
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Last updated 19 setembro 2024
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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
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