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

Abstract Background This cross-sectional study aimed to investigate the disease status, motor function, and medication treatment choices of genetically confirmed SMA patients. Methods A single-center, cross-sectional survey was conducted using web-based questionnaires and telephone interviews. Data...

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Bibliographic Details

Published in:	BMC Neurology
Main Authors:	Dingding Zhang, Fengxia Yao, Chao Ling, Shan Jiang, Xunzhe Yang, Yi Dai
Format:	Article
Language:	English
Published:	BMC 2025-10-01
Subjects:	Spinal muscular atrophy Disease-modifying therapies Nusinersen Risdiplam Treatment satisfaction
Online Access:	https://doi.org/10.1186/s12883-025-04441-9