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 …

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 …

Cystic fibrosis (CF) is a heritable, multiorgan disease that impacts all tissues that normally
express cystic fibrosis transmembrane conductance regulator (CFTR) protein. While the …

Abstract Elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA, Trikafta) is the newest Cystic Fibrosis
Transmembrane Conductance Regulator (CFTR) modulator drug approved by the Food and …

Cystic fibrosis (CF) has entered the era of variant-specific therapy, tailored to the genetic
variants in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene. CFTR …

CFTR modulators have dramatically changed the clinical course of CF in those fortunate
enough to receive them. Inevitably, randomised controlled trials during the development of …

Rationale: Elexacaftor, tezacaftor, and ivacaftor (ETI) in triple combination improves
pulmonary health for people with cystic fibrosis (PwCF). However, its impact on objective …

Small molecular modulators of the cystic fibrosis transmembrane conductance regulator
protein are transforming the care of people with cystic fibrosis. Highly effective modulators …

Highly-effective cystic fibrosis transmembrane conductance regulator (CFTR) modulator
therapy (HEMT) corrects the underlying molecular defect causing cystic fibrosis (CF) …

Introduction Reports of neuropsychiatric symptoms proximal to cystic fibrosis
transmembrane conductance regulator (CFTR) modulator initiation are emerging, but their …