今天3分钟我们来聊聊DMD细胞疗法3期结果：功能衰减减缓54%

Capricor公司细胞疗法Deramiocel治疗杜氏肌营养不良（DMD）的3期试验HOPE-3数据发表于《柳叶刀》，106例患者中，上肢功能衰减速度减缓54%，心脏功能也获改善。这是首个在晚期DMD人群中做出阳性结果的3期随机双盲试验，但尚未获批，8月22日将迎来FDA审批结果。参考文献（本期资料出处）：McDonald CM et al. Deramiocel heart-derived cellular therapy in advanced Duchenne muscular dystrophy (HOPE-3): a phase 3, randomised, double-blind, placebo-controlled trial. The Lancet, 2026. （2026-07-29 在线发表）Capricor Therapeutics. The Lancet Publishes HOPE-3 Data for Capricor Therapeutics' Deramiocel in Duchenne Muscular Dystrophy [新闻稿]. GlobeNewswire, 2026-07-29. 来源：https://www.globenewswire.com/news-release/2026/07/29/3335380/0/en/the-lancet-publishes-hope-3-data-for-capricor-therapeutics-deramiocel-in-duchenne-muscular-dystrophy.html生物通. 柳叶刀发表杜氏肌营养不良症的突破性临床试验结果, 2026-07-31. 来源：https://m.ebiotrade.com/newsf/2026-7/2026730172822379.htmA Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy (HOPE-3). ClinicalTrials.gov NCT05126758. 来源：https://clinicaltrials.ucsd.edu/trial/NCT05126758FDA Cellular, Tissue, and Gene Therapies Advisory Committee, July 29, 2026 Meeting Briefing Document.前往小宇宙评论区与主播互动