New therapies for spinal muscular atrophy: where we stand and what is next

The natural history of spinal muscular atrophy has been radically changed by the advent of improved standards of care and the availability of disease-modifying therapies. The aim of this paper is to provide the current therapeutic scenario including new perspectives and to report the challenges rela...

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Detalles Bibliográficos
Autores principales: Antonaci, Laura, Pera, Maria Carmela, Mercuri, Eugenio
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer Berlin Heidelberg 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10354145/
https://www.ncbi.nlm.nih.gov/pubmed/37067602
http://dx.doi.org/10.1007/s00431-023-04883-8