研究人员开展 CFTR 调节剂疗效对比研究，发现 VTD 和 ETI 对≥12 岁携带 F508del-CFTR-allele 的囊性纤维化患者更有效。 目的：旨在研究囊性纤维化跨膜传导调节因子（CFTR）调节剂，包括万赛扎考特 - 特扎考特 - 氘代维卡考特（VTD）、依来扎考特 - 特扎考特 - 依伐卡考 ...

Modulator drugs interact with mutant CFTR and force abnormally-folded proteins into a more functional structure. But these modulators are mutation-specific, and Skach estimates that 7% of cystic ...

Join Peter Grootenhuis of Vertex Pharmaceuticals as he delivers a brief overview of the drug discovery approaches and challenges to identify small molecules CFTR modulators. These compounds improve ...

More information: Raksha Jain et al, Impact of Cystic Fibrosis Transmembrane Conductance Regulator Modulators on Maternal ...

Other treatments may include antibiotics, pancreatic enzymes, a high-calorie diet, and newer generation drugs known as CFTR modulators. Severe cases may require a lung transplant. There is no cure ...

A study across 11 U.S. CF centers analyzed 307 pregnancies from 2010 to 2021, assessing the impact of CFTR modulators on pregnancy outcomes in individuals with cystic fibrosis.

The first CF rat model, CFTR tm1sage (termed CFTR-/- or KO), was designed in collaboration with SAGE (now Horizon Discovery) and subsequently characterized at UAB and distributed to the CF community.

A number of experimental techniques that involve measurements in CF patients are crucial to advancing CF science – including our understanding of basic aspects of disease pathogenesis, ...