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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...
| Autores principales: | , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
Springer Berlin Heidelberg
2023
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| 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 |