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Estimation Of The Quality Of Life Benefits Associated With Treatment For Spinal Muscular Atrophy
BACKGROUND: Spinal muscular atrophy (SMA) is a rare, genetic, progressive neuromuscular disorder characterized by severe muscle atrophy and weakness and is a leading genetic cause of death in infants and children. Nusinersen was the first treatment targeting the underlying cause of disease approved...
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| Publicado no: | Clinicoecon Outcomes Res |
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| Main Authors: | , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Dove
2019
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6818531/ https://ncbi.nlm.nih.gov/pubmed/31749625 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.2147/CEOR.S214084 |
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