History of development of the life-saving drug “Nusinersen” in spinal muscular atrophy

Spinal muscular atrophy (SMA) is an autosomal recessive disorder with an incidence of 1/6,000–1/10,000 and is the leading fatal disease among infants. Previously, there was no effective treatment for SMA. The first effective drug, nusinersen, was approved by the US FDA in December 2016, providing ho...

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Detalles Bibliográficos
Autores principales: Qiu, Jiaying, Wu, Liucheng, Qu, Ruobing, Jiang, Tao, Bai, Jialin, Sheng, Lei, Feng, Pengchao, Sun, Junjie
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2022
Materias:
Cellular Neuroscience
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9414009/
https://www.ncbi.nlm.nih.gov/pubmed/36035257
http://dx.doi.org/10.3389/fncel.2022.942976

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