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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...

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Detalles Bibliográficos
Autores principales: Ali, Imran, Gilchrist, Francis J, Carroll, William D, Alexander, John, Clayton, Sadie, Kulshrestha, Richa, Willis, Tracey, Samuels, Martin
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BMJ Publishing Group 2019
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