Spinal muscular atrophy: From approved therapies to future therapeutic targets for personalized medicine

Spinal muscular atrophy (SMA) is a devastating childhood motor neuron disease that, in the most severe cases and when left untreated, leads to death within the first two years of life. Recent therapeutic advances have given hope to families and patients by compensating for the deficiency in survival...

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Detalles Bibliográficos
Autores principales: Chaytow, Helena, Faller, Kiterie M.E., Huang, Yu-Ting, Gillingwater, Thomas H.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2021
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8324491/
https://www.ncbi.nlm.nih.gov/pubmed/34337562
http://dx.doi.org/10.1016/j.xcrm.2021.100346

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