PTC Therapeutics 的 PTC518 可降低亨廷顿舞蹈症患者的突变型亨廷顿蛋白,并在第 2 阶段 PIVOT-HD 研究中表现出安全性和耐受性。 PTC Therapeutics' PTC518 lowers mutant huntingtin protein in Huntington's disease patients, with safety and tolerability in Phase 2 PIVOT-HD study.
PTC Therapeutics 分享了 PTC518 治疗亨廷顿舞蹈症患者的第 2 阶段 PIVOT-HD 研究的中期结果,结果显示,第 12 个月时血液和脑脊液 (CSF) 中的突变型亨廷顿蛋白 (mHTT) 呈剂量依赖性降低。 PTC Therapeutics shares interim results from the Phase 2 PIVOT-HD study of PTC518 in Huntington's disease patients, showing dose-dependent lowering of mutant huntingtin (mHTT) protein in blood & cerebrospinal fluid (CSF) at Month 12. 总体运动评分(TMS)和综合统一亨廷顿氏病评定量表(cUHDRS)显示出良好的趋势。 Favorable trends demonstrated on Total Motor Score (TMS) & Composite Unified Huntington's Disease Rating Scale (cUHDRS). PTC518 仍然安全且耐受性良好。 PTC518 remains safe & well-tolerated.