ArticleNeurology and therapy2026
Impact of Early Intervention on Motor Milestone Achievement in Spinal Muscular Atrophy: Insights from Cure SMA Survey Data.
Article in Neurology and therapy, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
0 citing papers in PubMed.
No citing paper in PubMed yet.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
5 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
introductionThere is a paucity of real-world evidence on the impact of early treatment as well as polytherapy use on advanced motor milestone achievement in children with spinal muscular atrophy (SMA) with ≤ 2 survival motor neuron 2 gene (SMN2) copies. The objective of this study is to evaluate predictors of motor milestone change in children with ≤ 2 SMN2 copies, focusing on the timing of treatment initiation and the impact of treatment decisions.
methodsThis study employed a mixed longitudinal and repeated cross-sectional design. Data were drawn from the Cure SMA Community Update Survey, collected between 2020 and 2024. The study included 228 children with ≤ 2 SMN2 copies, each contributing at least one survey response, resulting in 414 total observations collected over the study period, with data provided by caregivers who reported residence in the USA. The primary outcome was maximum motor milestone achievement, evaluated using a mixed-effects ordinal hierarchical regression model to account for individual variability and repeated measures.
resultsTreatment before 3 weeks of age increased the odds of greater motor milestone achievement by 13.4-fold compared to treatment after 4 months (p = 0.001). SMA prenatal or newborn screening facilitated earlier treatment and reduced disparities in motor milestone achievement across income levels. Polytherapy was linked to 2.4-fold higher odds of reaching higher-level motor milestones compared to monotherapy (p = 0.01).
conclusionsEarly treatment initiation improved motor outcomes in children with ≤ 2 SMN2 copies. The findings support the importance of screening newborn screening as part of an effective strategy for optimizing motor development, as well as support the importance of further investigation into the relationship between the increasing proportion of polytherapy use and motor function development.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.