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 …

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 …

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 …

The autosomal recessive disease cystic fibrosis (CF) was once untreatable and deadly in
childhood, but now most patients survive to adulthood. Many countries have instituted CF …

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 …

Background Cystic fibrosis is an autosomal recessive disease caused by mutations in the
CFTR gene that lead to progressive respiratory decline. Some mutant CFTR proteins show …

Since the first description of cystic fibrosis in 1938, there have been significant advances in
both quality of life and longevity for people living with this disease. In this article we describe …

Background Though weight gain has been reported in some clinical trials of CFTR
modulators, the effect of elexacaftor-tezacaftor-ivacaftor on body weight, body mass index …

Salt and fluid absorption and secretion are two processes that are fundamental to epithelial
function and whole body fluid homeostasis, and as such are tightly regulated in epithelial …

Background Lumacaftor and ivacaftor combination treatment showed efficacy in patients
aged 12 years or older with cystic fibrosis homozygous for F508del-cystic fibrosis …