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Cognitive performance of adult patients with SMA before and after treatment initiation with nusinersen

BACKGROUND: Spinal muscular atrophy (SMA) is a genetic neuromuscular disease caused by mutations of the SMN1 gene. Deficient SMN protein causes irreversible degeneration of alpha motor neurons characterized by progressive muscle weakness and atrophy. Considering that SMA is a multi-systemic disorder...

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Detalles Bibliográficos
Autores principales: Vidovic, Maximilian, Freigang, Maren, Aust, Elisa, Linse, Katharina, Petzold, Daniel, Günther, René
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10243018/
https://www.ncbi.nlm.nih.gov/pubmed/37280513
http://dx.doi.org/10.1186/s12883-023-03261-z