For complex diseases, most drugs are highly ineffective, and the success rate of drug
discovery is in constant decline. While low quality, reproducibility issues, and translational …

Cystic fibrosis (CF) is a lethal inherited disease caused by mutations in the CF
transmembrane conductance regulator (CFTR) gene, which result in impairment of CFTR …

Based on its clinical benefits, Trikafta—the combination of folding correctors VX-661
(tezacaftor), VX-445 (elexacaftor), and the gating potentiator VX-770 (ivacaftor)—was FDA …

Rationale: The CFTR (cystic fibrosis transmembrane conductance regulator) modulator
combination elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) was shown to improve clinical …

Background Cystic fibrosis is a life-limiting disease that is caused by defective or deficient
cystic fibrosis transmembrane conductance regulator (CFTR) protein activity. Phe508del is …

The availability of the human genome sequence and tools for interrogating individual
genomes provide an unprecedented opportunity to apply genetics to medicine. Mendelian …

Positive results in pre-clinical studies of the triple combination of elexacaftor, tezacaftor and
ivacaftor, performed in airway epithelial cell cultures obtained from patients harbouring the …

The promise of gene therapy for the treatment of cystic fibrosis has yet to be fully clinically
realized despite years of effort toward correcting the underlying genetic defect in the cystic …

Advances over the past 25 years have revealed much about how the structural properties of
membranes and associated proteins are linked to the thermodynamics and kinetics of …

Identifying subjects with cystic fibrosis (CF) who may benefit from cystic fibrosis
transmembrane conductance regulator (CFTR)–modulating drugs is time-consuming, costly …