David Waltz

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: Evidence is provided that combination lumacaftor and ivACaftor improves FEV1 for patients with cystic fibrosis and safety (laboratory measurements and adverse events) is supported.

TL;DR: It is concluded that 28 days of tobramycin solution for inhalation of 300 mg twice daily is safe and effective for significant reduction of lower airway Pa density in young children with cystic fibrosis.