Results 11 to 20 of about 4,770 (180)

Efficacy and Safety of Elexacaftor-Tezacaftor-Ivacaftor in the Treatment of Cystic Fibrosis: A Systematic Review

Children, 2023
Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) is a new CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) modulator treatment, used over the last few years, which has shown an improvement in different clinical outcomes in patients with cystic ...
Nikoletta Kapouni, Maria Moustaki, Konstantinos Douros, Ioanna Loukou   +3 more
doaj   +1 more source

Quantitative Systems Toxicology Predicts Ivacaftor-Induced Oxidative Stress Contributes to CFTR Modulator Hepatotoxicity. [PDF]

Clin Pharmacol Ther
Cystic fibrosis (CF) is a chronic hereditary disease that affects tens of thousands of people worldwide. The introduction of CFTR modulator therapies such as elexacaftor/tezacaftor/ivacaftor (ETI) has significantly improved the quality of life of people with CF.
Shi A, Cornwell C, Yang K, Beringer PM.
europepmc   +2 more sources

Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Children 6 Through 11 Years of Age with Cystic Fibrosis Heterozygous for F508del and a Minimal Function Mutation: A Phase 3b, Randomized, Placebo-controlled Study [PDF]

, 2022
Rationale: The triple-combination regimen elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) was shown to be safe and efficacious in children aged 6 through 11 years with cystic fibrosis and at least one F508del-CFTR allele in a phase 3, open-label, single ...
Ahluwalia, Neil, Arteaga-Solis, Emilio, Brugha, Rossa, Bruinsma, Bote G, Davies, Jane C, Gartner, Silvia, Jennings, Mark, Legg, Julian, Mall, Marcus A, Moeller, Alexander, Mondejar-Lopez, Pedro, Moskowitz, Samuel M, Noel, Sabrina, Prais, Dario, Pressler, Tacjana, Ratjen, Felix, Reix, Philippe, Robinson, Paul D, Selvadurai, Hiran, Stehling, Florian, Tian, Simon, Wainwright, Claire E, Weinstock, Tanya G, Wu, Pan   +23 more
core   +1 more source

CFTR Modulators in People with Cystic Fibrosis: Real-World Evidence in France

Cells, 2022
Cystic fibrosis (CF) is a rare genetic multisystemic disease, the manifestations of which are due to mutations in the gene encoding the CF transmembrane conductance regulator (CFTR) protein and can lead to respiratory insufficiency and premature death ...
Lucile Regard, Clémence Martin, Espérie Burnet, Jennifer Da Silva, Pierre-Régis Burgel   +4 more
doaj   +1 more source

3D Automated Segmentation of Bronchial Abnormalities on Ultrashort Echo Time MRI: A Quantitative MR Outcome in Cystic Fibrosis. [PDF]

J Magn Reson Imaging
ABSTRACT Background Cystic fibrosis (CF) monitoring relies on computed tomography (CT), but ultra‐short echo time MRI (UTE‐MRI) offers a radiation‐free alternative. However, its clinical adoption is hindered by the laborious and subjective manual analysis, which prevents standardized quantification of bronchial abnormalities.
Bouzid AIH, Benlala I, de Senneville BD, Baldacci F, Macey J, Benhassen W, Su P, Bui S, Zysman M, Bustin A, Berger P, Dournes G.   +11 more
europepmc   +2 more sources

Simultaneous Quantification of Ivacaftor, Tezacaftor, and Elexacaftor in Cystic Fibrosis Patients’ Plasma by a Novel LC–MS/MS Method

Biomedicines, 2023
The new breakthrough cystic fibrosis (CF) drug combination of ivacaftor (IVA), tezacaftor (TEZ), and elexacaftor (ELX), namely “caftor” drugs, directly modulates the activity and trafficking of the defective CF transmembrane conductance regulator protein
Federica Pigliasco, Alessia Cafaro, Manuela Stella, Giammarco Baiardi, Sebastiano Barco, Nicoletta Pedemonte, Claudia D’Orsi, Federico Cresta, Rosaria Casciaro, Carlo Castellani, Maria Grazia Calevo, Francesca Mattioli, Giuliana Cangemi   +12 more
doaj   +1 more source

Gene Therapy: A Possible Alternative to CFTR Modulators?

Frontiers in Pharmacology, 2021
Cystic fibrosis (CF) is a rare genetic disease that affects several organs, but lung disease is the major cause of morbidity and mortality. The gene responsible for CF, the CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) gene, has been ...
J. Mercier, M. Ruffin, H. Corvol, H. Corvol, L. Guillot   +4 more
doaj   +1 more source

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

Regulatory T cell enhancement in adults with cystic fibrosis receiving Elexacaftor/Tezacaftor/Ivacaftor therapy

Frontiers in Immunology, 2023
IntroductionCystic fibrosis (CF), especially CF lung disease, is characterized by chronic infection, immune dysfunction including impairment of regulatory T cells (Tregs) and an exaggerated inflammatory response.
Dirk Westhölter, Jonas Raspe, Hendrik Uebner, Johannes Pipping, Mona Schmitz, Svenja Straßburg, Sivagurunathan Sutharsan, Matthias Welsner, Christian Taube, Sebastian Reuter   +9 more
doaj   +1 more source

The validated French CFAbd-Score reveals a lower burden of gastrointestinal symptoms in patients on Elexacaftor/Tezacaftor/Ivacaftor. [PDF]

J Pediatr Gastroenterol Nutr
Abstract Objectives Multiorgan abdominal involvement is a hallmark of Cystic fibrosis (CF). The CFAbd‐Score© is the first CF‐specific gastrointestinal patient reported outcome‐measure (PROM) developed following FDA‐guidelines. The PROM has proved to sensitively differentiate people with CF (pwCF) from healthy controls (HC).
Sermet-Gaudelus I, Sadrieh P, Bonnel AS, Enaud R, Wizla N, Languepin J, Nangui A, Deelawon A, Rave J, Dabelow S, Barucha A, Zagoya C, Duckstein F, Mainz JG.   +13 more
europepmc   +2 more sources