Longitudinal developmental profile of newborns and toddlers treated for spinal muscular atrophy

BACKGROUND: Spinal muscular atrophy (SMA) results from a loss-of-function mutation in the SMN1 gene. SMA patients suffer progressive motor disability, although no intellectual impairments have been described. Three drugs have been recently approved by the US Food and Drug Administration (FDA) and th...

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Detalles Bibliográficos
Autores principales: Ngawa, Magali, Dal Farra, Fabian, Marinescu, Andrei-Dan, Servais, Laurent
Formato: Online Artículo Texto
Lenguaje:English
Publicado: SAGE Publications 2023
Materias:
Original Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9944336/
https://www.ncbi.nlm.nih.gov/pubmed/36846472
http://dx.doi.org/10.1177/17562864231154335

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