Functional defect of variants in the adenosine triphosphate–binding sites of ABCB4 and their rescue by the cystic fibrosis transmembrane conductance regulator potentiator, ivacaftor (VX‐770)
Sign Up to like & getrecommendations! Published in 2017 at "Hepatology"
DOI: 10.1002/hep.28929
Abstract: ABCB4 (MDR3) is an adenosine triphosphate (ATP)‐binding cassette (ABC) transporter expressed at the canalicular membrane of hepatocytes, where it mediates phosphatidylcholine (PC) secretion. Variations in the ABCB4 gene are responsible for several biliary diseases, including… read more here.
Keywords: ivacaftor; ivacaftor 770; sites abcb4; potentiator ... See more keywords
CFTR‐dependent chloride efflux in cystic fibrosis mononuclear cells is increased by ivacaftor therapy
Sign Up to like & getrecommendations! Published in 2017 at "Pediatric Pulmonology"
DOI: 10.1002/ppul.23712
Abstract: The Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) potentiator ivacaftor (Kalydeco®) improves clinical outcome in G551D cystic fibrosis (CF) patients. Here, we have investigated whether ivacaftor has a clinical impact on non‐G551D gating mutations and function… read more here.
Keywords: ivacaftor; cystic fibrosis; fibrosis; cftr dependent ... See more keywords
Reversing the irreversible: Another potential benefit of CFTR modulators
Sign Up to like & getrecommendations! Published in 2020 at "Pediatric Pulmonology"
DOI: 10.1002/ppul.25047
Abstract: To the Editor, Cystic fibrosis (CF) is a genetic condition resulting from a fundamental defect in the cystic fibrosis transmembrane conductance regulator (CFTR) protein. This defect leads to a reduction or absence of chloride transport… read more here.
Keywords: ivacaftor; lumacaftor ivacaftor; cftr; age ... See more keywords
Repurposing Ivacaftor for treatment of Staphylococcus aureus infections.
Sign Up to like & getrecommendations! Published in 2017 at "International journal of antimicrobial agents"
DOI: 10.1016/j.ijantimicag.2017.03.020
Abstract: Drug repurposing of non-antimicrobials is a novel method to augment a seriously depleted drug pipeline for targeting drug-resistant pathogens. This article highlights the potent antimicrobial activity of Ivacaftor against Staphylococcus aureus, including vancomycin- and other… read more here.
Keywords: ivacaftor; repurposing ivacaftor; staphylococcus aureus; ivacaftor treatment ... See more keywords
Cystic fibrosis transmembrane conductance regulator (CFTR) modulators have differential effects on cystic fibrosis macrophage function
Sign Up to like & getrecommendations! Published in 2018 at "Scientific Reports"
DOI: 10.1038/s41598-018-35151-7
Abstract: Despite the addition of cystic fibrosis transmembrane conductance regulator (CFTR) modulators to the cystic fibrosis (CF) treatment regimen, patients with CF continue to suffer from chronic bacterial infections that lead to progressive respiratory morbidity. Host… read more here.
Keywords: ivacaftor; cystic fibrosis; macrophage function; macrophage ... See more keywords
The preclinical discovery and development of the combination of ivacaftor + tezacaftor used to treat cystic fibrosis
Sign Up to like & getrecommendations! Published in 2020 at "Expert Opinion on Drug Discovery"
DOI: 10.1080/17460441.2020.1750592
Abstract: ABSTRACT Introduction Cystic Fibrosis (CF) is caused by mutations in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene. The most common mutation, F508del, induces protein misprocessing and loss of CFTR function. The discovery through in… read more here.
Keywords: ivacaftor; cystic fibrosis; tezacaftor; cftr ... See more keywords
Tezacaftor and ivacaftor for the treatment of cystic fibrosis
Sign Up to like & getrecommendations! Published in 2019 at "Expert Review of Respiratory Medicine"
DOI: 10.1080/17476348.2020.1682998
Abstract: ABSTRACT Introduction: Cystic fibrosis (CF) is a complex, multi-system, genetic disease affecting over 70,000 people worldwide. The underlying defect is a mutation in the CFTR gene. Dysfunctional CFTR protein results in abnormal anion movement across… read more here.
Keywords: ivacaftor; cystic fibrosis; tezacaftor; cftr ... See more keywords
Ivacaftor for the treatment of cystic fibrosis in children under six years of age
Sign Up to like & getrecommendations! Published in 2020 at "Expert Review of Respiratory Medicine"
DOI: 10.1080/17476348.2020.1741352
Abstract: ABSTRACT Introduction Cystic fibrosis (CF) results from aberrant ion transport due to abnormalities or absence of the cystic fibrosis transmembrane conductance regulator (CFTR), a chloride transporter that resides on the apical surface of epithelial cells.… read more here.
Keywords: ivacaftor; cystic fibrosis; cftr; age ... See more keywords
Quantitative Method for the Analysis of Ivacaftor, Hydroxymethyl Ivacaftor, Ivacaftor Carboxylate, Lumacaftor, and Tezacaftor in Plasma and Sputum Using Liquid Chromatography With Tandem Mass Spectrometry and Its Clinical Applicability
Sign Up to like & getrecommendations! Published in 2020 at "Therapeutic Drug Monitoring"
DOI: 10.1097/ftd.0000000000000829
Abstract: Background: The novel cystic fibrosis transmembrane conductance regulator (CFTR) modulators, ivacaftor, lumacaftor, and tezacaftor, are the first drugs directly targeting the underlying pathophysiological mechanism in cystic fibrosis (CF); however, independent studies describing their pharmacokinetics are… read more here.
Keywords: lumacaftor tezacaftor; method; ivacaftor; plasma sputum ... See more keywords
Reduction of Recurrence Risk of Pancreatitis in Cystic Fibrosis With Ivacaftor: Case Series
Sign Up to like & getrecommendations! Published in 2018 at "Journal of Pediatric Gastroenterology and Nutrition"
DOI: 10.1097/mpg.0000000000001788
Abstract: ABSTRACT The effect of ivacaftor in patients with cystic fibrosis (CF) with recurrent pancreatitis is unknown. We conducted a multicenter retrospective study of patients with CF taking ivacaftor who had a history of recurrent pancreatitis.… read more here.
Keywords: ivacaftor; pancreatitis; reduction recurrence; cystic fibrosis ... See more keywords
The pharmacokinetic interaction between ivacaftor and ritonavir in healthy volunteers
Sign Up to like & getrecommendations! Published in 2017 at "British Journal of Clinical Pharmacology"
DOI: 10.1111/bcp.13324
Abstract: AIMS The aim of this study was to determine the pharmacokinetic interaction between ivacaftor and ritonavir. METHODS A liquid chromatography mass spectrometry (LC-MS) method was developed for the measurement of ivacaftor in plasma. An open-label,… read more here.
Keywords: ivacaftor; pharmacokinetic interaction; ivacaftor ritonavir; interaction ivacaftor ... See more keywords