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Safety, Tolerability, and Effect of Nusinersen in Non-ambulatory Adults With Spinal Muscular Atrophy

Objective: Investigation of the safety, tolerability, and treatment effect of nusinersen treatment in non-ambulatory adults with spinal muscular atrophy (SMA). Methods: Non-ambulatory individuals, aged 18 years or older with genetically confirmed 5q SMA were enrolled. In participants with spinal fus...

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Detalles Bibliográficos
Autores principales: Elsheikh, Bakri, Severyn, Steven, Zhao, Songzhu, Kline, David, Linsenmayer, Matthew, Kelly, Kristina, Tellez, Marco, Bartlett, Amy, Heintzman, Sarah, Reynolds, Jerry, Sterling, Gary, Weaver, Tristan, Rajneesh, Kiran, Kolb, Stephen J., Arnold, W. David
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8085528/
https://www.ncbi.nlm.nih.gov/pubmed/33935949
http://dx.doi.org/10.3389/fneur.2021.650532