Importance Cystic fibrosis, a genetic disorder defined by variants in the cystic fibrosis
transmembrane conductance regulator (CFTR) gene, affects more than 30 000 individuals in …

We are currently witnessing transformative change for people with cystic fibrosis with the
introduction of small molecule, mutation-specific drugs capable of restoring function of the …

Executive summary The past six decades have seen remarkable improvements in health
outcomes for people with cystic fibrosis, which was once a fatal disease of infants and young …

Background Reducing treatment burden is a priority for people with cystic fibrosis, whose
health has benefited from using new modulators that substantially increase CFTR protein …

This is the third in a series of four papers updating the European Cystic Fibrosis Society
(ECFS) standards for the care of people with CF. This paper focuses on recognising and …

Rationale: Previous studies showed that lumacaftor–ivacaftor therapy results in partial
rescue of CFTR (cystic fibrosis [CF] transmembrane conductance regulator) activity and a …

Background Hypertonic saline enhances mucociliary clearance and may lessen the
destructive inflammatory process in the airways. This is an update of a previously published …

Nitrogen multiple-breath washout is an established technique to assess functional residual
capacity and ventilation inhomogeneity in the lung. Accurate measurement of gas …

The growing use of modulator therapies aimed at restoring cystic fibrosis transmembrane
conductance regulator (CFTR) protein function in people with cystic fibrosis has …

Cystic fibrosis is an autosomal recessive genetic disease, mainly in Caucasian children and
young adults. It is caused by pathogenic variants in the CFTR (cystic fibrosis transmembrane …