Results 11 to 20 of about 3,103 (172)

Post-approval studies with the CFTR modulators Elexacaftor-Tezacaftor—Ivacaftor [PDF]

Frontiers in Pharmacology, 2023
Triple combination therapy with the CFTR modulators elexacaftor (ELX), tezacaftor (TEZ) and ivacaftor (IVA) has been qualified as a game changer in cystic fibrosis (CF).
Burkhard Tümmler, Burkhard Tümmler
doaj   +4 more sources

Successful pregnancy in a cystic fibrosis patient with a severe impairment of lung function receiving Elexacaftor-Tezacaftor-Ivacaftor [PDF]

Respiratory Medicine Case Reports, 2022
Before the arrival of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) modulators women with CF and impaired lung function were experiencing a high risk of complications and mortality during and the years after pregnancy.
Zisis Balmpouzis, Annabelle Faure van Rossum, David Baud, Alice Panchaud, Georgia Mitropoulou, Jesica Mazza Stalder, Angela Koutsokera   +6 more
doaj   +2 more sources

Elexacaftor-Tezacaftor-Ivacaftor: A Life-Changing Triple Combination of CFTR Modulator Drugs for Cystic Fibrosis

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
Mafalda Bacalhau, Mariana Camargo, Grace A. V. Magalhães-Ghiotto, Sybelle Drumond, Carlos Henrique M. Castelletti, Miquéias Lopes-Pacheco   +5 more
doaj   +2 more sources

Individualized approach to elexacaftor/tezacaftor/ivacaftor dosing in cystic fibrosis, in response to self-reported anxiety and neurocognitive adverse events: A case series [PDF]

Frontiers in Pharmacology, 2023
The prevalence of mental health disorders is high among people with Cystic Fibrosis. The psychological symptoms in CF are associated with poor adherence, worse treatment outcomes, and greater health utilization/cost.
Hisham Ibrahim, Hisham Ibrahim, Hammad Danish, David Morrissey, David Morrissey, Kevin F. Deasy, Kevin F. Deasy, Mairead McCarthy, James Dorgan, Claire Fleming, Ciara Howlett, Sarah Twohig, Tamara Vagg, Tamara Vagg, Desmond M. Murphy, Desmond M. Murphy, Michael Maher, Michael Maher, Barry J. Plant, Barry J. Plant   +19 more
doaj   +2 more sources

Organic Synthesis and Current Understanding of the Mechanisms of CFTR Modulator Drugs Ivacaftor, Tezacaftor, and Elexacaftor

Molecules
The monogenic rare disease Cystic Fibrosis (CF) is caused by mutations in the gene encoding the CF transmembrane conductance (CFTR) protein, an anion channel expressed at the apical plasma membrane of epithelial cells.
Filipa C. Ferreira, Camilla D. Buarque, Miquéias Lopes-Pacheco   +2 more
doaj   +2 more sources

Elexacaftor-Tezacaftor-Ivacaftor Treatment Reduces Abdominal Symptoms in Cystic Fibrosis-Early results Obtained With the CF-Specific CFAbd-Score

Frontiers in Pharmacology, 2022
Background: The novel and highly effective CFTR modulator combination of elexacaftor-tezacaftor-ivacaftor (ETI) has been shown to improve lung function and body weight in people with Cystic Fibrosis (pwCF) carrying a F508del mutation. However, the impact
Jochen G. Mainz, Jochen G. Mainz, Carlos Zagoya, Louise Polte, Lutz Naehrlich, Lutz Naehrlich, Lenny Sasse, Olaf Eickmeier, Christina Smaczny, Anton Barucha, Lilith Bechinger, Franziska Duckstein, Ludwik Kurzidim, Ludwik Kurzidim, Patience Eschenhagen, Laura Caley, Daniel Peckham, Daniel Peckham, Carsten Schwarz   +18 more
doaj   +1 more source

Elexacaftor is a CFTR potentiator and acts synergistically with ivacaftor during acute and chronic treatment

Scientific Reports, 2021
Cystic fibrosis (CF) is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR), which lead to early death due to progressive lung disease. The development of small-molecule modulators that directly interact with CFTR to aid
Ciaran A. Shaughnessy, Pamela L. Zeitlin, Preston E. Bratcher   +2 more
doaj   +1 more source

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

Novel CFTR modulator combinations maximise rescue of G85E and N1303K in rectal organoids

ERJ Open Research, 2022
Introduction Cystic fibrosis (CF) is a severe monogenic disorder caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
Marjolein M. Ensinck, Liesbeth De Keersmaecker, Anabela S. Ramalho, Senne Cuyx, Stephanie Van Biervliet, Lieven Dupont, Frauke Christ, Zeger Debyser, François Vermeulen, Marianne S. Carlon   +9 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