$信立泰(SZ002294)$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...
A Phase 2a Study of TPN-101 in Patients With Amyotrophic Lateral Sclerosis (ALS) and/or Frontotemporal Dementia (FTD) Associated With Hexanucleotide Repeat Expansion in the C9orf72 Gene (C9ORF72 ALS/FTD) This is a Phase 2a study to assess the the safety and tolerability of TPN-101 in pat...