Review of CFTR modulators 2020.
Sign Up to like & getrecommendations! Published in 2021 at "Pediatric pulmonology"
DOI: 10.1002/ppul.25627
Abstract: Cystic 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… read more here.
Keywords: modulators 2020; cystic fibrosis; cftr modulators; cftr ... See more keywords
The impact of CFTR modulators on bone health
Sign Up to like & getrecommendations! Published in 2023 at "Pediatric Pulmonology"
DOI: 10.1002/ppul.26314
Abstract: We are hopeful that CTFR modulation does have a positive impact of bone health, but we have concerns about this study design being used to support the hypothesis This article is protected by copyright. All… read more here.
Keywords: impact cftr; bone health; impact; cftr modulators ... See more keywords
Beyond the 10%: Unraveling the genetic diversity in Turkish cystic fibrosis patients not eligible for CFTR modulators
Sign Up to like & getrecommendations! Published in 2024 at "Pediatric Pulmonology"
DOI: 10.1002/ppul.27181
Abstract: Cystic fibrosis (CF) is an autosomal recessive disease caused by variants of CFTR gene. Over 2000 variants have been identified, and new drugs called CFTR modulators have been developed to target specific defects in the… read more here.
Keywords: unraveling genetic; beyond unraveling; cystic fibrosis; eligible cftr ... See more keywords
Development and clinical implementation of an LC-HRMS method for ivacaftor, lumacaftor, tezacaftor and elexacaftor in human plasma and breast milk
Sign Up to like & getrecommendations! Published in 2024 at "Analytical and Bioanalytical Chemistry"
DOI: 10.1007/s00216-024-05496-2
Abstract: The four cystic fibrosis (CF) transmembrane conductance regulator (CFTR) modulators, ivacaftor, lumacaftor, tezacaftor, and elexacaftor, have revolutionised the treatment of CF by direct action on the protein target behind the disease’s development. The aim was… read more here.
Keywords: lumacaftor tezacaftor; tezacaftor elexacaftor; breast milk; milk ... See more keywords
Airway microbial diversity is decreased in young children with cystic fibrosis compared to healthy controls but improved with CFTR modulation
Sign Up to like & getrecommendations! Published in 2020 at "Heliyon"
DOI: 10.1016/j.heliyon.2020.e04104
Abstract: Background Culture-independent next generation sequencing has identified diverse microbial communities within the cystic fibrosis (CF) airway. The study objective was to test for differences in the upper airway microbiome of children with CF and healthy… read more here.
Keywords: cystic fibrosis; cftr modulators; diversity; healthy controls ... See more keywords
Discovery and Development of CFTR Modulators for the Treatment of Cystic Fibrosis.
Sign Up to like & getrecommendations! Published in 2025 at "Journal of medicinal chemistry"
DOI: 10.1021/acs.jmedchem.4c02547
Abstract: Cystic fibrosis (CF) is a genetic disorder caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which regulates ion and fluid transport across epithelial cells. Mutations lead to complications, with life-limiting lung… read more here.
Keywords: chemistry; discovery development; cftr; cystic fibrosis ... See more keywords
Molecular Prosthetics and CFTR Modulators Additively Increase Secretory HCO3- Flux in Cystic Fibrosis Airway Epithelia.
Sign Up to like & getrecommendations! Published in 2025 at "ACS chemical biology"
DOI: 10.1021/acschembio.5c00473
Abstract: Cystic fibrosis (CF) is caused by loss-of-function mutations in the gene encoding the cystic fibrosis transmembrane conductance regulator (CFTR), an anion channel predominantly expressed on the apical membrane of epithelial cells. Reduced Cl- and HCO3-… read more here.
Keywords: function; airway epithelia; cftr; cystic fibrosis ... See more keywords
A Systematic Review of the Clinical Efficacy and Safety of CFTR Modulators in Cystic Fibrosis
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DOI: 10.1038/s41598-019-43652-2
Abstract: Several placebo-controlled trials have been recently published evaluating novel therapies targeting the defective CFTR protein. This systematic review examines the clinical efficacy and safety of CFTR modulators in individuals with cystic fibrosis (CF) with specific… read more here.
Keywords: cftr modulators; clinical efficacy; cftr; efficacy safety ... See more keywords
Discovery of CFTR modulators for the treatment of cystic fibrosis
Sign Up to like & getrecommendations! Published in 2021 at "Expert Opinion on Drug Discovery"
DOI: 10.1080/17460441.2021.1912732
Abstract: ABSTRACT Introduction Cystic fibrosis (CF) is a life-threatening inherited disease caused by mutations in the gene encoding the CF transmembrane conductance regulator (CFTR) protein, an anion channel expressed at the apical membrane of secretory epithelia.… read more here.
Keywords: cftr; cftr modulators; cystic fibrosis; discovery cftr ... See more keywords
Cystic fibrosis transmembrane conductance receptor modulator therapy in cystic fibrosis, an update.
Sign Up to like & getrecommendations! Published in 2020 at "Current Opinion in Pediatrics"
DOI: 10.1097/mop.0000000000000892
Abstract: PURPOSE OF REVIEW Cystic fibrosis transmembrane conductance receptor (CFTR) modulators are a new class of drugs that treat the underlying cause of cystic fibrosis. To date, there are four approved medications, which are mutation-specific. Although… read more here.
Keywords: modulator therapy; cystic fibrosis; fibrosis; cftr modulators ... See more keywords
To treat or not to treat: CFTR modulators after lung transplantation
Sign Up to like & getrecommendations! Published in 2021 at "Pediatric Transplantation"
DOI: 10.1111/petr.14007
Abstract: Evidence for the extrapulmonary benefits of (CFTR) modulators is rapidly expanding. The use of CFTR modulators in CF patients who have undergone lung transplantation is not clear without guidance published in the medical literature to… read more here.
Keywords: transplantation treat; transplantation; cftr modulators; lung transplantation ... See more keywords