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

• मुख्य लेखकों: Francis J Gilchrist (लेखक), William D Carroll (लेखक), Tracey Willis (लेखक), Martin Samuels (लेखक), Imran Ali (लेखक), John Alexander (लेखक), Sadie Clayton (लेखक), Richa Kulshrestha (लेखक)
• स्वरूप: पुस्तक
• प्रकाशित: BMJ Publishing Group, 2019-12-01T00:00:00Z.
• विषय: article
• ऑनलाइन पहुंच: Connect to this object online.

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...