European Respiratory Journal ( IF 16.6 ) Pub Date : 2024-11-14 Britto, C. J., Taylor-Cousar, J. L.
Since the original characterisation of cystic fibrosis (CF), its treatment has focused primarily on mitigating its most severe manifestations. However, in the past decade, the emergence of therapies that improve cystic fibrosis transmembrane conductance regulator (CFTR) protein processing and function has been changing the lives of those eligible for these small molecule therapies, first in the USA in 2012 [1] and, later, in other, mostly high-income, countries. However, the widespread use of CFTR modulators has highlighted significant disparities in access that have extended beyond biology and genetics to challenges in affordability and access to care.
中文翻译:
直到它为所有人完成:CFTR 调节剂标签扩展的作用
自囊性纤维化 (cystic fibrosis, CF) 最初表征以来,其治疗主要集中在减轻其最严重的表现上。然而,在过去十年中,改善囊性纤维化跨膜传导调节因子 (CFTR) 蛋白加工和功能的疗法的出现一直在改变那些有资格接受这些小分子疗法的人的生活,首先是 2012 年的美国 [1],后来是其他主要高收入国家。然而,CFTR 调节剂的广泛使用凸显了在可及性方面的显着差异,这些差异已经超越了生物学和遗传学,延伸到可负担性和获得护理方面的挑战。