$信立泰(SZ002294)$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...
SEQ ID NO. 1 shows the complete amino acid sequence (163 residues) ofVibrio choleraelipoprotein 15 (VcLp15) as retrievable from the public database UniProt, accession no. Q9KQN6. Amino acid residues 1-25 constitute the signal sequence; the mature VcLp15 comprises amino acid residues 26-...
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TPN-101 had lowering effects on key biomarkers of neurodegeneration and neuroinflammation, including neurofilament light chain (NfL) and interleukin 6 (IL-6) Treatment with TPN-101 also showed clinical benefits on the Revised ALS Functional Rating Scale (ALSFRS-R) and Vital Capacity, key clinical ...