近日,Transposon宣布其在研逆转录酶抑制剂TPN-101治疗肌萎缩侧索硬化(ALS)患者2期试验的最终结果,这些患者的C9orf72基因具有六核苷酸重复扩增(C9orf72相关的ALS)。数据显示,TPN-101治疗可显著降低ALS疾病进展,并减缓患者的肺活量(VC)下降情形,公司计划将TPN-101推进至用以治疗C9orf72相关ALS患者的3期研究。(来源:T...
至第48周,两组肺活量下降幅度都小于自然病程。 在双盲阶段,至24周,TPN-101组与安慰剂组相比《ALS功能评分量表(ALSFRS-R)》评分下降幅度相当(平均下降7.2 vs 6.7)。在随后的开放标签阶段,TPN-101组24周ALSFRS-R评分只下降了一半。至第48周,两组ALSFRS-R评分下降幅度都小于自然病程。 TPN-101 是逆转录转座子LI...
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