Observational studyDevelopmental medicine and child neurology2025
Bulbar function in children with spinal muscular atrophy type 1 treated with nusinersen.
Observational study in Developmental medicine and child neurology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
3 citing papers in PubMed.
- Ten years of disease-modifying therapy in spinal muscular atrophy: lessons learned and future directions.Nature reviews. Neurology · 2026Review
- Oral and Swallowing Abilities Tool (OrSAT) in Individuals with Type I SMA Older than 24 Months: A Pilot Study.Children (Basel, Switzerland) · 2026Article
- Bulbar function in children with spinal muscular atrophy type 1 treated with nusinersen.Developmental medicine and child neurology · 2025Observational
Corrections and comments
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Authors and funding
10 authors.
Funding
Abstract
aimTo describe bulbar function trajectories in patients with spinal muscular atrophy (SMA) type 1 treated with nusinersen in the UK and Italy.
methodIn 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 (interquartile range = 4.1-14.4 months).]
resultsThe Italian cohort had more participants in the 1b group (symptom onset >2 weeks and <3 months), while the UK had more participants in the 1c group (symptom onset <6 months). Over 2 years, the p-FOIS/CEDAS captured lack of bulbar improvement in the 1b group, with 40% displaying stability and 45% showing decline; in the 1c group, stability was captured (71%). OrSAT captured improvement in 47% of the 1b group and 43% of the 1c group at 2 years; this was predominantly because of age-related speech acquisition and feeding viscosities, where the item was not age-appropriate at baseline.
interpretationThe p-FOIS/CEDAS and OrSAT measures capture complementary information on the effect of disease-modifying treatments (DMTs) on bulbar function. Further studies are required to understand bulbar function trajectories in symptomatic and presymptomatic cohorts with SMA receiving different DMTs.
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