Safety, Tolerability, and Effect of Nusinersen Treatment in Ambulatory Adults With 5q-SMA

Objective: To determine the safety and tolerability of nusinersen treatment in ambulatory adults with spinal muscular atrophy (SMA) and investigate the treatment effect on muscle strength, physical function, and motor unit physiology.Methods: Individuals aged 18 years or older with genetically confi...

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Bibliographic Details
Main Authors: Bakri Elsheikh, Steven Severyn, Songzhu Zhao, David Kline, Matthew Linsenmayer, Kristina Kelly, Marco Tellez, Amy Bartlett, Sarah Heintzman, Jerry Reynolds, Gary Sterling, Tristan Weaver, Kiran Rajneesh, Stephen J. Kolb, W. David Arnold
Format: Article
Language:English
Published: Frontiers Media S.A. 2021-05-01
Series:Frontiers in Neurology
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Online Access:https://www.frontiersin.org/articles/10.3389/fneur.2021.650535/full