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September 10, 2025Orphanet Journal of Rare DiseasesOpen Access

Longitudinal efficacy of risdiplam treatment in Chinese children with spinal muscular atrophy

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Authors

YYYue YanDZDifeng ZhuKMKai Ma

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Overview

Observational analysis revealed significant motor function improvement in children with SMA treated with risdiplam, indicating effective outcomes.

Key Points

  • Significant improvement in motor function was observed in children with SMA treated with risdiplam, highlighting its efficacy.
  • The CHOPINTEND score increased by 2.7 points/month in SMA type 1 patients, providing concrete evidence of treatment impact.
  • Generalized estimating equation analysis assessed motor function improvements of various scales, strengthening the validity of results.
  • Risdiplam demonstrated a good safety profile, with pneumonia as the most common adverse event reported.

Cite This Study

Yan et al. (2025) studied this question.

synapsesocial.com/papers/68c23e94b210217d647932f3https://doi.org/10.1186/s13023-025-03929-4
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