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Therapy development for spinal muscular atrophy: perspectives for muscular dystrophies and neurodegenerative disorders
BACKGROUND: Major efforts have been made in the last decade to develop and improve therapies for proximal spinal muscular atrophy (SMA). The introduction of Nusinersen/Spinraza™ as an antisense oligonucleotide therapy, Onasemnogene abeparvovec/Zolgensma™ as an AAV9-based gene therapy and Risdiplam/E...
Autores principales: | , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
BioMed Central
2022
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8725368/ https://www.ncbi.nlm.nih.gov/pubmed/34983696 http://dx.doi.org/10.1186/s42466-021-00162-9 |