Rare Diseases Research: Clinical Trial for Niemann-Pick Type C
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Niemann-Pick disease type C
NIH clinical trial begins for treatment of rare, fatal neurological disorder
When Drug Development for Rare Disease Hit Setback, Parents Were Stung - WSJ
Combination therapy a potential strategy for treating Niemann-Pick disease
Valley family fights for drug to save sick child
Adult Onset Niemann-Pick type C (NPC) – NNPDF
FDA rejection of rare disease drug leads Orphazyme to cut two thirds of staff - MedCity News
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
Niemann–Pick Disease Type C
Rare Disease/Orphan Disease
PDF) Consensus clinical management guidelines for Niemann-Pick disease type C
Niemann Pick Disease Type C - Symptoms, Causes, Treatment
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