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 …

Cystic fibrosis (CF) is an inherited multisystemic disease that can cause progressive
bronchiectasis, pancreatic endocrine and exocrine insufficiency, distal intestinal obstruction …

Background Elexacaftor plus tezacaftor plus ivacaftor is a triple-combination cystic fibrosis
transmembrane conductance regulator (CFTR) modulator regimen shown to be generally …

Background: Pulmonary rehabilitation is recognized as a core component of the
management of individuals with chronic respiratory disease. Since the 2006 American …

Objective: The purpose of this study was to quantify the effect of physical activity (in both
descriptive and intervention studies) on health-related quality of life (HRQOL) in children …

Background Increasing the activity of defective cystic fibrosis transmembrane conductance
regulator (CFTR) protein is a potential treatment for cystic fibrosis. Methods We conducted a …

Rationale: Ivacaftor is a cystic fibrosis transmembrane conductance regulator (CFTR)
potentiator recently approved for patients with CF age 6 and older with the G551D mutation …

Background The phe508del CFTR mutation causes cystic fibrosis by limiting the amount of
CFTR protein that reaches the epithelial cell surface. We tested combination treatment with …

Background Ivacaftor is used to treat patients with CF and a G551D gating mutation; the
KONNECTION study assessed the efficacy and safety of ivacaftor in patients with CF and a …

Background A new approach in the treatment of cystic fibrosis involves improving the
function of mutant cystic fibrosis transmembrane conductance regulator (CFTR). VX-770, a …