Onasemnogene abeparvovec in spinal muscular atrophy: an Australian experience of safety and efficacy
Abstract Objective To provide a greater understanding of the tolerability, safety and clinical outcomes of onasemnogene abeparvovec in real‐world practice, in a broad population of infants with spinal muscular atrophy (SMA). Methods A prospective cohort study of children with SMA treated with onasem...
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| Principais autores: | , , , , , , , , , , , , , |
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| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Wiley
2022-03-01
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| coleção: | Annals of Clinical and Translational Neurology |
| Acesso em linha: | https://doi.org/10.1002/acn3.51519 |
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