Nusinersen in adult patients with 5q spinal muscular atrophy: A multicenter observational cohorts' study

BACKGROUND AND PURPOSE: The aim was to assess the safety and efficacy of nusinersen in adult 5q spinal muscular atrophy (SMA) patients. METHODS: Patients older than 15 years and followed for at least 6 months with one motor scale (Hammersmith Functional Motor Scale Expanded, HFMSE; Revised Upper Lim...

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Detalles Bibliográficos
Autores principales: Vázquez‐Costa, Juan F., Povedano, Mónica, Nascimiento‐Osorio, Andrés E., Moreno Escribano, Antonio, Kapetanovic Garcia, Solange, Dominguez, Raul, Exposito, Jessica M., González, Laura, Marco, Carla, Medina Castillo, Julita, Muelas, Nuria, Natera de Benito, Daniel, Ñungo Garzón, Nancy Carolina, Pitarch Castellano, Inmaculada, Sevilla, Teresa, Hervás, David
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2022
Materias:
Child Neurology
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9796289/
https://www.ncbi.nlm.nih.gov/pubmed/35872571
http://dx.doi.org/10.1111/ene.15501

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