早期治疗是 SMA 患儿的生命线。
Early treatment is a lifeline for infants with SMA.
机构信息
Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, MD, USA.
Department of Neuroscience, Johns Hopkins University School of Medicine, Baltimore, MD, USA.
出版信息
Nat Med. 2022 Jul;28(7):1348-1349. doi: 10.1038/s41591-022-01889-x.
Abstract
In the Phase 3 SPRINT trial, pre-symptomatic gene therapy demonstrated impressive clinical outcomes in infants with a genetic diagnosis of SMA; long-term safety follow-up of these patients must now be a key priority.
摘要
在第 3 阶段的 SPRINT 试验中，症状前基因治疗在患有 SMA 遗传诊断的婴儿中显示出令人印象深刻的临床结果；这些患者的长期安全性随访现在必须成为一个关键重点。
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