Observational studyJournal of neurology2025
Upper limb motor function in individuals with SMA type 2: natural history and impact of therapies.
Observational study in Journal of neurology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.
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Who cites it
1 citing paper in PubMed.
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Authors and funding
25 authors.
Funding
Abstract
objectivesTo describe the natural history of the upper limb motor function in spinal muscular atrophy (SMA) type 2 and analyze the impact of SMA-modifying therapies on Revised Upper Limb Module (RULM) scores.
methodsThis is a retrospective, multicenter, observational study including individuals with SMA type 2, aged between 30 months and 20 years at the time of their first RULM assessment, with available follow-up data.
resultsWe enrolled 149 untreated individuals as part of the natural history cohort, with a mean age of 9.5 years at the first assessment and a mean follow-up duration of 3.98 years (SD 1.97, range 0.3-7.7). An increase in RULM scores was observed in early childhood within this cohort. However, after 4.4 years of age, a gradual decline in RULM scores was noted with increasing age. In contrast, RULM scores were significantly higher in individuals receiving treatment with nusinersen or risdiplam compared to natural history data.
conclusionOur findings provide a natural history of upper extremity motor function in children and adolescents with SMA type 2. The RULM scores typically improve during the early years of life, peaking around 4.4 years of age, after which they progressively decline with age. The data presented here will facilitate the assessment of treatment response in individuals with SMA type 2, especially in those with already severely limited motor function.
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