Healthcare utilisation in children with SMA type 1 treated with nusinersen: a single centre retrospective review

• Main Authors: Francis J Gilchrist (Author), William D Carroll (Author), Tracey Willis (Author), Martin Samuels (Author), Imran Ali (Author), John Alexander (Author), Sadie Clayton (Author), Richa Kulshrestha (Author)
• Format: Bog
• Udgivet: BMJ Publishing Group, 2019-12-01T00:00:00Z.
• Fag: article
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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 util...