Results 31 to 40 of about 1,459,532 (261)
Rapid therapeutic advances in CFTR modulator science [PDF]
Pediatric Pulmonology, 2018 AbstractCystic fibrosis (CF) is an autosomal recessive genetic disease caused by variants in the gene encoding the cystic fibrosis transmembrane conduction regulator (CFTR) protein. Loss of CFTR function disrupts chloride, bicarbonate and regulation of sodium transport, producing a cascade of mucus obstruction, inflammation, pulmonary infection, and ...
John P Clancy
exaly +3 more sources
Progress in precision medicine in cystic fibrosis: a focus on CFTR modulator therapy
Breathe, 2021 The genetic multisystem condition cystic fibrosis (CF) has seen a paradigm shift in therapeutic approaches within the past decade. Since the first clinical descriptions in the 1930s, treatment advances had focused on the downstream consequences of a ...
D. Tewkesbury, R. Robey, P. Barry
semanticscholar +2 more sources
Review of CFTR modulators 2020 [PDF]
Pediatric Pulmonology, 2021 AbstractCystic fibrosis transmembrane conductance regulator (CFTR) modulators are small molecules that directly impact the CFTR protein, improving the function of the CFTR chloride and bicarbonate channel. Beginning in 2012 with the Food and Drug Administration approval of the first CFTR modulator, ivacaftor, this class of medications has had largely ...
Danielle M. Goetz, Adrienne P. Savant
openaire +2 more sources
Journal of the Belgian Society of Radiology, 2022 Objectives: Cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapy effects on respiratory function, pulmonary exacerbations and quality of life have been well documented. However, CFTR modulator therapy effects on sinus disease have
Corrado Tagliati +10 more
doaj +1 more source
Personalizing CFTR modulator therapies [PDF]
, 2021 over 2000 genetic mutations that can cause CF, resulting in many phenotypes. Recently new drugs were developed that treat the disease at the origin of the problem; they enhance the function of the Cystic Fibrosis Transmembrane conductance Regulator (CFTR)
Berkers, Gitte
openaire +2 more sources
Pharmacological Responses of the G542X-CFTR to CFTR Modulators
Frontiers in Molecular Biosciences, 2022 Cystic fibrosis (CF) is a lethal hereditary disease caused by loss-of-function mutations of the chloride channel cystic fibrosis transmembrane conductance regulator (CFTR). With the development of small-molecule CFTR modulators, including correctors that facilitate protein folding and expression and potentiators that promote channel activity, about 90%
Xinxiu Fang +3 more
openaire +3 more sources
Pharmaceuticals, 2023 Cystic fibrosis (CF) is a potentially fatal monogenic disease that causes a progressive multisystemic pathology. Over the last decade, the introduction of CF transmembrane conductance regulator (CFTR) modulator drugs into clinical practice has profoundly
M. Bacalhau +5 more
semanticscholar +1 more source
Tezacaftor/Ivacaftor therapy has negligible effects on the cystic fibrosis gut microbiome
Microbiology Spectrum, 2023 People with cystic fibrosis (pwCF) experience a range of persistent gastrointestinal symptoms throughout life. There is evidence indicating interaction between the microbiota and gut pathophysiology in CF.
Ryan Marsh +7 more
doaj +1 more source
NIHR Open Research, 2023 Background: Gastrointestinal symptoms in cystic fibrosis (CF) are common and intrusive to daily life. Relieving gastrointestinal symptoms was identified as an important research priority and previously explored in an international survey in 2018. However,
Vinuja Premakumar +12 more
doaj +1 more source