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Pre-symptomatic risdiplam treatment for spinal muscular atrophy initiated at 12 days post-birth: 14-month safety and developmental outcomes.

IF 2 4区 医学 Q2 PEDIATRICS
Pediatric Investigation Pub Date : 2025-04-14 eCollection Date: 2025-06-01 DOI:10.1002/ped4.70003
Zhihao Sun, Liping Wu, Yufan Hui, Yuhang Zhong, Jiangtao Yang, Zhiyong Sun, Xiaoling Huang, Hui Xiong
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出生后12天开始对脊髓性肌萎缩症进行症状前利斯平治疗：14个月的安全性和发育结局
本文章由计算机程序翻译，如有差异，请以英文原文为准。
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来源期刊
Pediatric Investigation
Pediatric Investigation Medicine-Pediatrics, Perinatology and Child Health
CiteScore
3.30
自引率
0.00%
发文量
176
审稿时长
12 weeks
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