EPISODE · Jan 15, 2026 · 5 MIN
今天3分钟我们来聊聊B型血友病基因治疗终获长期答案
from 源于细胞BioInsid的个人播客
NEJM发表的HOPE-B研究最终分析显示，B型血友病基因治疗Etranacogene dezaparvovec一次给药后，患者凝血因子IX活性长期维持，年出血率显著下降，多数患者停止或减少常规替代治疗，实现功能性治愈可能，安全性可控。 前往小宇宙评论区与主播互动
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今天3分钟我们来聊聊B型血友病基因治疗终获长期答案
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