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  • Publication : 14 07 2019
  • Catégorie :La recherche médicale et génétique

Nusinersen Improves Walking Distance and Reduces Fatigue in Later-Onset SMA.    

INTRODUCTION: Ambulatory individuals with spinal muscular atrophy (SMA) experience muscle weakness, gait impairments and fatigue that affect their walking ability. Improvements have been observed in motor function in children treated with nusinersen but its impact on fatigue has not been studied.
METHODS: Post hoc analyses examined changes in 6-Minute Walk Test (6MWT) distance and fatigue in children and adolescents with SMA Type II and III who received their first dose of nusinersen in the phase Ib/IIa open-label CS2 study and were ambulatory during CS2 or the extension study CS12.
RESULTS: Fourteen children performed the 6MWT. Median (25th, 75th percentile) distance walked increased over time by 98.0 (62.0, 135.0) meters at day 1050, while median fatigue changed by -3.8% (-19.7%, 1.4%).
DISCUSSION: These results support previous studies demonstrating clinically meaningful effects of nusinersen on motor function in children and adolescents with later-onset SMA. This article is protected by copyright. All rights reserved.

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