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Assessing Bulbar Function in Spinal Muscular Atrophy Using Patient-Reported Outcomes

BACKGROUND: Novel Spinal Muscular Atrophy (SMA) treatments have demonstrated improvements on motor measures that are clearly distinct from the natural history of progressive decline. Comparable measures are needed to monitor bulbar function, which is affected in severe SMA. OBJECTIVE: To assess bulb...

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Detalles Bibliográficos
Autores principales: Dunaway Young, Sally, Pasternak, Amy, Duong, Tina, McGrattan, Katlyn E., Stranberg, Sarah, Maczek, Elizabeth, Dias, Courtney, Tang, Whitney, Parker, Dana, Levine, Alexis, Rohan, Alyssa, Wolford, Connie, Martens, William, McDermott, Michael P., Darras, Basil T., Day, John W.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: IOS Press 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10258884/
https://www.ncbi.nlm.nih.gov/pubmed/36776075
http://dx.doi.org/10.3233/JND-221573