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 …

Recurrent and chronic bacterial infections are common in people with cystic fibrosis (CF)
and contribute to lung function decline. Antibiotics are the mainstay in the treatment of …

Rationale: Elexacaftor–tezacaftor–ivacaftor is a CFTR (cystic fibrosis [CF] transmembrane
conductance regulator) modulator combination, developed for patients with CF with at least …

Importance Race and ethnicity are routinely used to inform pulmonary function test (PFT)
interpretation. However, there is no biological justification for such use, and it may reinforce …

Background The European Medicines Agency has approved the cystic fibrosis
transmembrane conductance regulator (CFTR) modulator combination …

Background Elexacaftor-tezacaftor-ivacaftor induces rapid clinical improvement in patients
with cystic fibrosis (CF) and advanced pulmonary disease, often leading to suspend the …

Cystic fibrosis is a genetic disease with multisystem involvement and associated morbidity
and mortality that are most directly related to progressive lung disease. The hallmark …

Rationale: Limited data exist on the safety and effectiveness of elexacaftor-tezacaftor-
ivacaftor (ETI) in people with cystic fibrosis (pwCF) and advanced lung disease. Objectives …

Cystic fibrosis (CF) may cause a spectrum of hepatobiliary complications, including portal
hypertension, multilobular cirrhosis, and liver failure. Current guidelines on the detection …

Cystic fibrosis (CF) is a genetic disease in which mutations in the gene encoding for the CF
transmembrane conductance regulator protein result in a multisystem disease dominated by …