Abstract
CFTR (cystic fibrosis transmembrane conductance regulator) modulators
are small molecules that directly change the CFTR protein, improving
function of the CFTR chloride channel. Beginning in 2012 with the FDA
approval of the first CFTR modulator, ivacaftor, this class of
medication has had largely positive effects on many outcomes in people
with cystic fibrosis (pwCF), including lung function, quality of life,
and growth. There have been continued exciting developments in the
current research on CFTR modulators, expanding beyond original studies.
This first part of a three-part Cystic Fibrosis (CF) Year in Review 2020
will focus on research on CFTR modulators. Subsequent parts of the CF
year in review will cover pulmonary and infectious inflammatory aspects,
and the multisystem effects of CF in the 2020 literature. The review
focuses on articles from Pediatric Pulmonology, but it includes articles
from other journals that are of particular interest to clinicians. New
developments in CF research continue to be brought forth to the CF
community, deepening the understanding of this disease and improving
clinical care.