Defining When Nusinersen Starts to Work: Time to Clinical Benefit in Patients with SMA Types 1–3 from a Real-World Cohort in China

<b>Background</b>: 5q spinal muscular atrophy (SMA) is a hereditary neuromuscular disorder characterized by progressive muscle weakness. Nusinersen, the first disease-modifying therapy for SMA, has demonstrated efficacy in both clinical trials and real-world studies. However, the precise timing of t...

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Bibliografiske detaljer

Principais autores:	Ying Wu, Shuang Li, Yanbin Fan, Yuan Wu, Jie Zhang, Hui Dong, Yao Zhang, Xiaoling Yang, Hui Xiong, Cuijie Wei
Format:	Artigo
Sprog:	Inglês
Udgivet:	MDPI AG 2026-06-01
Serier:	Diagnostics
Fag:	spinal muscular atrophy Nusinersen efficacy safety real world
Online adgang:	https://www.mdpi.com/2075-4418/16/12/1828
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