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Whole‐blood dysregulation of actin‐cytoskeleton pathway in adult spinal muscular atrophy patients

OBJECTIVE: Recent advances in therapeutics have improved prognosis for severely affected spinal muscular atrophy (SMA) type 1 and 2 patients, while the best method of treatment for SMA type 3 patients with later onset of disease is unknown. To better characterize the SMA type 3 population and provid...

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Detalles Bibliográficos
Autores principales: Siranosian, Jennifer J., Nery, Flavia C., Alves, Christiano R. R., Siranosian, Benjamin A., Lyons, Nicholas J., Eichelberger, Eric J., Garner, Reid, Da Silva Duarte Lepez, Salomé, Johnstone, Alec J., Subramanian, Aravind, Swoboda, Kathryn J.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7359125/
https://www.ncbi.nlm.nih.gov/pubmed/32558393
http://dx.doi.org/10.1002/acn3.51092