To prevent irreversible organ damage, an infant with early-onset Pompe’s disease was treated in utero; early data suggest improved outcomes and safety of this approach.

中文翻译：

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庞贝氏病的产前治疗


为了防止不可逆的器官损伤，一名患有早发庞贝氏症的婴儿在子宫内接受了治疗。早期数据表明这种方法的结果和安全性有所改善。