Bulbar function in children with spinal muscular atrophy type 1 treated with nusinersen.

ABSTRACT:

Aim

To describe bulbar function trajectories in patients with spinal muscular atrophy (SMA) type 1 treated with nusinersen in the UK and Italy.

Method

In two previously reported, retrospective, observational cohort studies, we observed the 2-year change in the Children's Eating and Drinking Ability Scale (CEDAS) (the revised and optimized version of the Paediatric Functional Oral Intake Scale [p-FOIS]) and Oral and Swallowing Ability Tool (OrSAT) in 44 patients treated on average at 8.3 months (interquartile range = 4.1-14.4 months), with data collected every 6 months from treatment initiation. [Correction added on 30 June 2025 after first online publication: In the preceding sentence, 11.2 months (interquartile range = 4.1-24.7 months) has been changed to 8.3 months (interq

SUBMITTER: Stimpson G 

PROVIDER: S-EPMC12618960 | biostudies-literature | 2025 Dec

REPOSITORIES: biostudies-literature