Article

Effectiveness and Safety of Risdiplam in Chinese Adolescents and Adults with Spinal Muscular Atrophy: A Prospective Real-World Study

2025-11-26

Abstract excerpt

<title>Abstract</title> <p>Background Evidence for the efficacy of risdiplam in adolescents and adults with 5q spinal muscular atrophy (SMA) has been demonstrated in clinical trials, but its sustained effectiveness and safety in real-world settings, particularly in Chinese populations, remains to be demonstrated. We evaluated the efficacy and safety of risdiplam in this population in a real-world setting. Methods...

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Literature Corpus work: 2ab0a837-224b-5cab-bf6b-4f211850e478
DOI: 10.21203/rs.3.rs-7966590/v1

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