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October 8, 2025BMC NeurologyOpen Access

Disease characteristics and treatment status of genetically confirmed spinal muscular atrophy patients: a cross-sectional survey in China

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Authors

DZDingding ZhangPeking Union Medical College HospitalFYFengxia YaoChinese Academy of Medical Sciences & Peking Union Medical CollegeCLChao LingChinese Academy of Medical Sciences & Peking Union Medical College

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Overview

Cross-sectional survey reveals treatment trends and motor function in SMA patients, indicating the need for better education on DMTs.

Key Points

  • Motor function was generally well-maintained, with 51.9% of SMA patients able to sit independently despite high prevalence of spinal deformities.
  • Among 106 participants, 66.0% had used nusinersen and 24.5% had used risdiplam, highlighting diverse treatment regimens in SMA.
  • Data were collected through a cross-sectional survey using web-based questionnaires and telephone interviews with genetically diagnosed SMA patients.
  • Improving patient education about DMTs is essential, as 48.8% expressed treatment dissatisfaction over perceived efficacy and costs.

Cite This Study

Zhang et al. (2025) studied this question.

synapsesocial.com/papers/68e6860af44b9035634c2016https://doi.org/10.1186/s12883-025-04441-9
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Also Consider

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