Rationale: As the life expectancy for individuals with cystic fibrosis (CF) continues to
improve, an emphasis on optimizing health-related quality of life (HRQoL) has become …

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 …

Cystic fibrosis (CF) is a rare, progressive, multi-organ genetic disease. Ivacaftor, a small-
molecule CF transmembrane conductance regulator modulator, was the first medication to …

Background Lung delivery of plasmid DNA encoding the CFTR gene complexed with a
cationic liposome is a potential treatment option for patients with cystic fibrosis. We aimed to …

Background Patients with cystic fibrosis (CF) have increasing treatment complexity and high
treatment burden. We describe trends in treatment complexity and evaluate its relationship …

The care for individuals with cystic fibrosis (CF) with at least one F508del mutation will
greatly change as a result of the unparalleled clinical benefits observed with the new triple …

Background The cystic fibrosis transmembrane conductance regulator (CFTR) potentiator,
ivacaftor, was first approved for people with CF and the G551D CFTR mutation. This study …

Background Cystic fibrosis (CF) is a life shortening disease, however prognosis has
improved and the adult population is growing. Most adults with cystic fibrosis live …

Introduction: The CFTR modulator drug elexacaftor/tezacaftor/ivacaftor (ETI) was shown to
improve CFTR function and clinical symptoms in patients with cystic fibrosis (CF) with at …

Rationale: The lung clearance index (LCI) is responsive to acute respiratory events in
preschool children with cystic fibrosis (CF), but its utility to identify and manage these events …