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Lessons from a negative gene therapy trial for Duchenne muscular dystrophy
Nature Medicine ( IF 58.7 ) Pub Date : 2024-10-09 , DOI: 10.1038/s41591-024-03316-9
Simone Spuler, Gian Domenico Borasio, Ulrike Grittner

Delandistrogene moxeparvovec received FDA approval after a negative trial, which highlights the many complexities and challenges of drug development in this setting.

中文翻译:


杜氏肌营养不良症阴性基因治疗试验的经验教训



Delandistrogene moxeparvovec 在一次否定试验后获得了 FDA 的批准,这凸显了在这种情况下药物开发的许多复杂性和挑战。
更新日期:2024-10-09
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