Rare Diseases Research: Clinical Trial for Niemann-Pick Type C
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Efficacy and safety of arimoclomol in Niemann‐Pick disease type C: Results from a double‐blind, randomised, placebo‐controlled, multinational phase 2/3 trial of a novel treatment - Mengel - 2021 - Journal of Inherited
The Niemann-Pick Foundation finances a clinical trial of a drug to stop the cognitive deterioration of the disease – IDIBELL
Fig 1., Subcortical Volumetric Reductions in Adult Niemann-Pick Disease Type C: A Cross-Sectional Study
Speaker Series - Niemann-Pick Disease Type C
Niemann–Pick type C disease: cellular pathology and pharmacotherapy - Wheeler - 2020 - Journal of Neurochemistry - Wiley Online Library
Miglustat in Niemann-Pick disease type C patients: a review, Orphanet Journal of Rare Diseases
Niemann-Pick disease: Causes, symptoms, treatment, and more
Immune dysfunction in Niemann‐Pick disease type C - Platt - 2016 - Journal of Neurochemistry - Wiley Online Library
Adult Onset Niemann-Pick type C (NPC) – NNPDF
Valley family fights for drug to save sick child
Estimated prevalence of Niemann–Pick type C disease in Quebec
Rare Diseases Research: Clinical Trial for Niemann-Pick Type C
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por adulto (o preço varia de acordo com o tamanho do grupo)
