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Long-term safety and efficacy of patisiran for hereditary transthyretin-mediated amyloidosis with polyneuropathy: 12-month results of an open-label extension study

Archive ouverte | Adams, David | CCSD

International audience. BACKGROUND : Hereditary transthyretin-mediated amyloidosis is a rare, inherited, progressive disease caused by mutations in the transthyretin (TTR) gene. We assessed the safety and efficacy o...

Motor unit number index correlates with disability in Charcot-Marie-Tooth disease

Archive ouverte | Bas, Joachim | CCSD

International audience. Objective: The aim of this study was to assess the usefulness of motor unit number index (MUNIX) tech-nique in Charcot-Marie-Tooth disease and test the correlation between MUNIX and clinical ...

Combination of serum and CSF neurofilament-light and neuroinflammatory biomarkers to evaluate ALS

Archive ouverte | Brodovitch, Alexandre | CCSD

International audience. This monocentric prospective study of patient suffering from Amyotrophic lateral sclerosis (ALS) aims to evaluate the prognosis and diagnostic potential of both Neurofilament-Light (Nf-L) and...

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