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“It’s a vote for hope”: first gene therapy for muscular dystrophy nears approval, but will it work?
Nature ( IF 50.5 ) Pub Date : 2023-06-02 , DOI: 10.1038/d41586-023-01799-z
Sara Reardon

The FDA’s decision, expected this month, follows several setbacks and delays and will pose difficult choices for the families of children with Duchenne muscular dystrophy.

中文翻译:


“这是对希望的投票”:第一个针对肌肉萎缩症的基因疗法即将获得批准,但它会起作用吗?



FDA 的决定预计于本月做出,该决定经历了数次挫折和拖延,这将为杜氏肌营养不良症儿童的家庭带来艰难的选择。
更新日期:2023-06-03
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