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Healthcare utilisation in children with SMA type 1 treated with nusinersen: a single centre retrospective review
BACKGROUND: Nusinersen has been used to treat spinal muscular atrophy type 1 (SMA1) in the UK since 2017. While initial trials showed neuromuscular benefit from treating SMA1, there is little information on the respiratory effects of nusinersen. We aimed to look at the respiratory care, hospital uti...
Autores principales: | , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
BMJ Publishing Group
2019
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6937078/ https://www.ncbi.nlm.nih.gov/pubmed/31909224 http://dx.doi.org/10.1136/bmjpo-2019-000572 |