Patients with Spinal Muscular Atrophy Type 1 Achieve and Maintain Bulbar Function Following Onasemnogene Abeparvovec Treatment(1)

BACKGROUND: Improvement and maintenance of bulbar function are goals of disease-modifying treatments for spinal muscular atrophy (SMA). Lack of standardized measures and a widely accepted definition of bulbar function represents a gap in SMA care. OBJECTIVE: A multidisciplinary team conducted post-h...

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Detalles Bibliográficos
Autores principales: McGrattan, Katlyn E., Shell, Richard D., Hurst-Davis, Rebecca, Young, Sally Dunaway, O’Brien, Eamonn, Lavrov, Arseniy, Wallach, Shiri, LaMarca, Nicole, Reyna, Sandra P., Darras, Basil T.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: IOS Press 2023
Materias:
Research Report
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10357176/
https://www.ncbi.nlm.nih.gov/pubmed/37092232
http://dx.doi.org/10.3233/JND-221531

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10357176/
https://www.ncbi.nlm.nih.gov/pubmed/37092232
http://dx.doi.org/10.3233/JND-221531

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