… and development of the combination of ivacaftor + tezacaftor used to treat cystic fibrosis …
More precise and affordable direct and surrogate biomarkers are also needed to better gauge …

… The disease is caused by mutations in the cystic fibrosis transmembrane conductance
regulator (CFTR) gene which encodes the CFTR protein, a protein kinase A-activated ATP-gated …

… CF has clearly taught us that the first step to precision medicine is to understand molecular
pathology, and we are appreciating that within a specific chILD gene, there may be multiple …

… These recommendations were informed by the observation in the Cystic Fibrosis Foundation
… with cystic fibrosis: Cystic Fibrosis Foundation consensus guidelines. J Cyst Fibros 2019;18:…

Cystic fibrosis (CF) is a lethal, recessive, genetic disease affecting approximately 1 in 2500
live births among Caucasians. The CF gene codes for a cAMP/PKA‐dependent, ATP‐…

Cystic fibrosis (CF) is a life-shortening monogenic disease caused by mutations in the gene
encoding the CF transmembrane conductance regulator (CFTR) protein, an anion channel …

Treatment, prognosis, and quality of life for people with cystic fibrosis (CF) have improved …
The recent improvement in outcomes is attributable to the advent of cystic fibrosis …

… Recent breakthroughs in cystic fibrosis (CF) transmembrane conductance regulator (CFTR)-directed
therapies have heralded a new era of precision medicine for patients with CF [1,2,3,…

… Cystic fibrosis (CF) is the leading genetic disease among Caucasians; however, advances
in diagnosis and treatment have improved both quality and quantity of life for those affected. A …

… Other limitations to the precision of our estimates include the novelty of triple combination
treatment meaning negotiations and drug rollout is ongoing in some settings, alongside …