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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...
Autores principales: | , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
John Wiley and Sons Inc.
2022
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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 |