Population-based assessment of nusinersen efficacy in children with spinal muscular atrophy: a 3-year follow-up study

Nusinersen (Spinraza®) improves survival of infants with hereditary proximal spinal muscular atrophy and motor function in children up to 12 years. Population-based assessments of treatment efficacy are limited and confined to select cohorts of patients. We performed a nationwide, population-based,...

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Detalles Bibliográficos
Autores principales: Scheijmans, Féline E V, Cuppen, Inge, van Eijk, Ruben P A, Wijngaarde, Camiel A, Schoenmakers, Marja A G C, van der Woude, Danny R, Bartels, Bart, Veldhoen, Esther S, Oude Lansink, Irene L B, Groen, Ewout J N, Asselman, Fay-Lynn, Wadman, Renske I, van der Pol, W Ludo
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2022
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9651026/
https://www.ncbi.nlm.nih.gov/pubmed/36382221
http://dx.doi.org/10.1093/braincomms/fcac269

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