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Cystic fibrosis: triple drug regimens that target defective ion channel improve lung function, studies show
The BMJ ( IF 105.7 ) Pub Date : 2018-10-19 , DOI: 10.1136/bmj.k4377
Susan Mayor

Triple drug combinations that improve the activity of the cystic fibrosis transmembrane conductance regulator (CFTR), the anion channel that is defective or deficient in the condition, significantly improve lung function in patients with cystic fibrosis who have the most common CFTR gene mutations, two early stage randomised trials have reported.12
“These reports represent a major breakthrough in cystic fibrosis therapeutics, with the potential for improving health and possibly survival in all patients who carry the most common CFTR mutation,” said Fernando Holguin of the University of Colorado in Aurora, USA, in an accompanying editorial.3
Both trials evaluated the efficacy and safety of one of two new generation, small molecule CFTR correctors, VX-659 and VX-445. These compounds …


中文翻译:

囊性纤维化:针对不良离子通道的三类药物疗法可改善肺功能

三联药物组合可改善囊性纤维化跨膜电导调节剂(CFTR)的活性,该通道不良或缺乏,可显着改善具有最常见CFTR基因突变的囊性纤维化患者的肺功能,这是两个早期阶段阶段随机试验已报道。1 2
“这些报告代表了囊性纤维化治疗方法的重大突破,具有改善所有携带最常见CFTR突变的患者的健康和可能生存的潜力,”美国奥罗拉科罗拉多大学的Fernando Holguin说道。随附社论。3
两项试验均评估了两种新一代小分子CFTR校正剂VX-659和VX-445之一的功效和安全性。这些化合物……
更新日期:2018-10-19
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