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“It’s a vote for hope”: first gene therapy for muscular dystrophy nears approval, but will it work?
Nature ( IF 64.8 ) 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:

“这是希望的一票”:首个肌营养不良症基因疗法即将获得批准,但它会奏效吗?

FDA 的决定预计将于本月发布,但在经历了几次挫折和延误之后,将给患有杜氏肌营养不良症的儿童家庭带来艰难的选择。
更新日期:2023-06-03
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