Sarepta Therapeutics· @Sarepta · X·· 2026-03-25AI 评分42
AI 导读
今天我们公布了SRP-1001用于FSHD1和SRP-1003用于DM1的1/2期递增剂量研究的早期临床结果。结果显示剂量依赖性肌肉暴露、早期生物标志物效应和良好的耐受性，增强了我们对平台差异化递送能力的信心。了解更多：https://bit.ly/4buZc1c
正文
Today we announced early clinical results from Phase 1/2 ascending-dose studies of SRP-1001 for FSHD1 and SRP-1003 for DM1. The results demonstrated dose-dependent muscle exposure, early biomarker effects and favorable tolerability, reinforcing confidence in the potential for differentiated delivery capabilities of our platform. Learn more: https://bit.ly/4buZc1c
来源：Sarepta Therapeutics · x.com