Tag: ivacaftor

1. In this multicenter, phase 3 study of cystic fibrosis patients who were previously unresponsive to modulator regimens, triple-combination therapy with the next-generation corrector elexacaftor resulted in a significantly improved percentage of predicted forced expiratory volume in one second versus placebo after 4 and 24 weeks. 2. Improvements were also...
1. In this multicenter, phase 3 study of cystic fibrosis patients who were previously unresponsive to modulator regimens, triple-combination therapy with the next-generation corrector elexacaftor resulted in a significantly improved percentage of predicted forced expiratory volume in one second versus placebo after 4 and 24 weeks. 2. Improvements were also...
1. A G551D missense mutation in the cystic fibrosis transmembrane conductance receptor (CFTR) is a genetic cause of cystic fibrosis (CF). 2. In utero administration of the CFTR-potentiating drug, VX-770 (ivacaftor) in a ferret genetic model of cystic fibrosis provided protection from CF pathologies in the pancreas, intestine, and male...