Cargando…

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...

Descripción completa

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:
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