Charlotte M. McKee

TL;DR: These data show that lumacaftor in combination with ivacaftors provided a benefit for patients with cystic fibrosis homozygous for the Phe508del CFTR mutation.

TL;DR: Elexacaftor-tezacaft or-ivacaft or was efficacious in patients with cystic fibrosis with Phe508del-minimal function genotypes, in whom previous CFTR modulator regimens were ineffective.

TL;DR: Elexacaftor plus tezacaftorplus ivacaftors provided clinically robust benefit compared with tezacftor plus ivACaftor alone, with a favourable safety profile, and shows the potential to lead to transformative improvements in the lives of people with cystic fibrosis who are homozygous for the F508del mutation.

TL;DR: The use of VX‐445–tezacaftor–ivacaftor to target Phe508del CFTR protein resulted in increased CFTR function in vitro and translated to improvements in patients with cystic fibrosis with one or two Phe48del alleles.

TL;DR: CFTR modulator therapy with tezacaftor–ivacaftor or ivacaftors alone was efficacious in patients with cystic fibrosis who were heterozygous for the Phe508del deletion and a CFTR residual‐function mutation.