ArticleFrontiers in neurology
Longitudinal motor function and biomarker correlates in treated adult spinal muscular atrophy: a single-center cohort study.
Article in Frontiers in neurology. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Abstract
Background: Long-term clinical trajectories and accessible biomarkers in adult spinal muscular atrophy (SMA) remain insufficiently characterized under real-world treatment conditions. We conducted a single-center longitudinal study to evaluate motor function, genotype-phenotype relationships, and clinically accessible biomarkers in treated adults with SMA. Methods: Twenty-three Japanese adults with genetically confirmed 5q-SMA treated with nusinersen or risdiplam (2018-2025) underwent repeated assessments of Revised Upper Limb Module (RULM), Hammersmith Functional Motor Scale Expanded (HFMSE), and laboratory, physiological, and electrophysiological measures. Associations were explored using correlation analyses, linear mixed-effects models, and exploratory machine-learning approaches. Results: Higher Conclusion: In treated adults with SMA, simple clinical measures such as %VC, CK, ulnar CMAP amplitude are associated with motor status and may support routine monitoring, while prediction of treatment responsiveness remains challenging.
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