参考资料：[1] FDA Approves First Gene Therapy for Treatment of Certain Patients with Duchenne Muscular Dystrophy. Retrieved June 22, 2023 from https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-treatment-certain-patients-duchenne-muscular-dystrophy[2] Sarepta Therapeutics to Share Expression and Safety Results from Study 103 (ENDEAVOR) Evaluating SRP-9001, its Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy. Retrieved May 23, 2023, from https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-share-expression-and-safety-results-study?_ga=2.161787388.1980422535.1621350865-784075460.1620058440[3] Study SRP-9001-103: 12-Week Expression and Safety Data Using Commercially Representative Material. Retrieved May 23, 2023, from https://investorrelations.sarepta.com/static-files/09358a6f-98f9-4e11-a59e-db6b0d0f584f[4] Sarepta Therapeutics’ Investigational Gene Therapy SRP-9001 for Duchenne Muscular Dystrophy Demonstrates Significant Functional Improvements Across Multiple Studies, Retrieved July 7th, 2022, from https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-investigational-gene-therapy-srp-9001[5] FDA approves Sarepta’s Duchenne muscular dystrophy gene therapy via accelerated pathway. Retrieved June 22, 2023 from https://endpts.com/fda-approves-sareptas-duchenne-muscular-dystrophy-gene-therapy-via-accelerated-pathway/ 免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。 版权说明：本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。