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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Discussion

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

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.

Zhang et al. (2025) studied this question.

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