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Evolution of bulbar function in spinal muscular atrophy type 1 treated with nusinersen

AIM: To assess the evolution of bulbar function in nusinersen‐treated spinal muscular atrophy type 1 (SMA1). METHOD: This single‐centre retrospective study identified 24 patients (14 females and 10 males) with SMA1, treated with nusinersen between 2017 and 2020. We adapted and validated the Paediatr...

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Detalles Bibliográficos
Autores principales:	Weststrate, Harriet, Stimpson, Georgia, Thomas, Lily, Scoto, Mariacristina, Johnson, Emily, Stewart, Alexandra, Muntoni, Francesco, Baranello, Giovanni, Conway, Eleanor
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	John Wiley and Sons Inc. 2022
Materias:	Original Articles
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9306995/ https://www.ncbi.nlm.nih.gov/pubmed/35103306 http://dx.doi.org/10.1111/dmcn.15171

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