Administration practices of and adherence to nusinersen in children with spinal muscular atrophy: a multicenter disease registry study in China

Administration practices of and adherence to nusinersen in children with spinal muscular atrophy: a multicenter disease registry study in China

Abstract Background Nusinersen was the first approved disease modifying therapy (DMT) for spinal muscular atrophy (SMA). Intrathecal administration of nusinersen enables drug delivery directly to the central nervous system, where the motor neurons are located. Per the package insert, individuals wit...

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Main Authors: Jing Peng, Xiaoli Yao, Rong Luo, Xiuxia Wang, Liwen Wu, Jianmin Zhong, Ruifeng Jin, Xinguo Lu, Jianmin Liang, Siqi Hong, Lin Yang, Xiaoli Zhang, Shanshan Mao, Zhe Tao, Jun Hu, Dan Sun, Hua Wang, Li Zhang, Yanyan Xia, Ken Chen, Yi Wang
Format: Article
Language:English
Published: BMC 2025-03-01
Series:BMC Pediatrics
Subjects:
Nusinersen
Children
Registry
Adherence
Administration practices
Online Access:https://doi.org/10.1186/s12887-024-05290-0
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https://doi.org/10.1186/s12887-024-05290-0

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