Healthcare utilisation in children with SMA type 1 treated with nusinersen: a single centre retrospective review.
(2019)
Journal Article
Gilchrist. (2019). Healthcare utilisation in children with SMA type 1 treated with nusinersen: a single centre retrospective review. BMJ Paediatrics Open, https://doi.org/10.1136/bmjpo-2019-000572
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 a... Read More about Healthcare utilisation in children with SMA type 1 treated with nusinersen: a single centre retrospective review..