Results 11 to 20 of about 6,532 (197)

In Vitro Restoration of Colistin Susceptibility by Ivacaftor Synergy with Limited Reproducibility in a Murine Pneumonia Model [PDF]

Antibiotics
Background: We aimed to investigate the potential synergistic effect of ivacaftor combined with colistin against Pseudomonas aeruginosa and Klebsiella pneumoniae, and to elucidate the underlying molecular mechanisms through metabolomic analysis and its ...
Ana Verónica Halperin, Franziska Schwartz, Lars Christophersen, José Pérez-del Palacio, Manuel Ponce-Alonso, José Avendaño-Ortiz, Juan de Dios Caballero, Rafael Cantón, Claus Moser, Rosa del Campo   +9 more
doaj   +2 more sources

Transcriptomic Responses to Ivacaftor and Prediction of Ivacaftor Clinical Responsiveness

American Journal of Respiratory Cell and Molecular Biology, 2019
Abstract Ivacaftor is a drug that was recently approved by the U.S. Food and Drug Administration for the treatment of patients with cystic fibrosis (CF) and at least one copy of the G511D mutation in the CFTR (CF transmembrane conductance regulator) gene. The transcriptomic effect of ivacaftor in patients with CF remains unclear. Here,
Zhe Sun, Yale Jiang, Annabel A Ferguson   +2 more
exaly   +4 more sources

Synthesis and Evaluation of Ivacaftor Derivatives with Reduced Lipophilicity

, 2023
Mutations in the unique ATP-binding cassette anion channel, the cystic fibrosis conductance regulator (CFTR), lead to the inherited fatal disease known as cystic fibrosis (CF).
Russell D. Viirre (2440522), Gagan D. Gupta (12326583), Saujanya Acharya (17450996), Roxana Sühring (2810290), Melissa Iazzi (14497938), Phillip Junor (17450993), Jitesh Doshi (11886037)   +6 more
core   +4 more sources

Management of Infants Exposed to Prenatal and Postnatal Elexacaftor/Tezacaftor/Ivacaftor: A Cross-Sectional Survey. [PDF]

Health Sci Rep
Health Science Reports, Volume 9, Issue 5, May 2026.
Haskett H, Ren CL.
europepmc   +2 more sources

Calibration and validation of modeled 5-year survival predictions among people with cystic fibrosis treated with the cystic fibrosis transmembrane conductance regulator modulator ivacaftor using United States registry data.

PLoS ONE, 2023
ObjectivesCystic fibrosis (CF) is a rare genetic disease characterized by life-shortening lung function decline. Ivacaftor, a CF transmembrane conductance regulator modulator (CFTRm), was approved in 2012 for people with CF with specific gene mutations ...
Lisa J McGarry, Zahra Bhaiwala, Andrea Lopez, Conor Chandler, Christopher G Pelligra, Jaime L Rubin, Theodore G Liou   +6 more
doaj   +3 more sources

A Comparative Analysis of Pricing and Reimbursement of Cystic Fibrosis Transmembrane Conductance Regulator Modulators in Europe

Frontiers in Pharmacology, 2021
Objectives: Cystic fibrosis transmembrane conductance regulator (CFTR) modulators, Kalydeco® (ivacaftor), Orkambi® (lumacaftor/ivacaftor) and Symkevi® (tezacaftor/ivacaftor), have substantially improved patients’ lives yet significantly burden healthcare
Khadidja Abdallah, Kris De Boeck, Marc Dooms, Steven Simoens   +3 more
doaj   +1 more source

Utility of Fecal Elastase-1 in Estimating Exocrine Pancreatic Function in Cystic Fibrosis: A Scoping Review. [PDF]

Pediatr Pulmonol
ABSTRACT Background In people with cystic fibrosis (pwCF), identification of exocrine pancreatic insufficiency (EPI) is essential to prevent steatorrhea and, if not managed actively, can lead to catastrophic consequences. Fecal elastase‐1 (FE‐1) is a widely used test to screen for EPI in cystic fibrosis (CF).
Sankararaman S, Hendrix S, Neudecker M, Borowitz D.   +3 more
europepmc   +2 more sources

Effects of CFTR modulators on serum biomarkers of liver fibrosis in children with cystic fibrosis

Hepatology Communications, 2023
The cystic fibrosis (CF) transmembrane conductance regulator corrector/potentiator combinations lumacaftor/ivacaftor and elexacaftor/tezacaftor/ivacaftor improve sweat chloride, pulmonary function, and nutrition.
Steven Levitte, Yonathan Fuchs, Russell Wise, Zachary M. Sellers   +3 more
doaj   +1 more source

Improved Quality of Life in Children With Cystic Fibrosis Who Received Transmembrane Conductance Regulator Modulators. [PDF]

Acta Paediatr
ABSTRACT Aim Children with cystic fibrosis (CF) face substantial daily treatment burdens and the effects of transmembrane conductance regulator modulators on these have not been sufficiently described. We evaluated changes in treatment burden after elexacaftor tezacaftor ivacaftor (ETI) was initiated.
Svedberg M, Lundqvist F, Östrand HA, Krantz C, Ekman P, Imberg H.   +5 more
europepmc   +2 more sources

Physiologically Based Pharmacokinetic Modeling of CFTR Modulation in People with Cystic Fibrosis Transitioning from Mono or Dual Regimens to Triple-Combination Elexacaftor/Tezacaftor/Ivacaftor

Pulmonary Therapy, 2020
Introduction The triple-combination (TC) cystic fibrosis transmembrane conductance regulator (CFTR) modulator regimen elexacaftor, tezacaftor, and ivacaftor was shown to be safe and efficacious in phase 3 trials of people with cystic fibrosis (pwCF) ≥ 12 
Alice Tsai, Shu-Pei Wu, Eric Haseltine, Sanjeev Kumar, Samuel M. Moskowitz, Paul Panorchan, Kushal Shah   +6 more
doaj   +1 more source