An open-label extension study of ivacaftor in children with CF and a CFTR gating mutation initiating treatment at age 2-5 years (KLIMB).
Therapeutic benefit observed with the CFTR potentiator, ivacaftor, in a CF patient homozygous for the W1282X CFTR nonsense mutation
Ivacaftor and symptoms of extra-oesophageal reflux in patients with cystic fibrosis and G551D mutation
CFTR potentiator therapy ameliorates impaired insulin secretion in CF patients with a gating mutation
