Results 21 to 30 of about 3,103 (172)

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

Sustained effectiveness of elexacaftor-tezacaftor-ivacaftor in lung transplant candidates with cystic fibrosis

, 2022
International audienceBackground: Elexacaftor-tezacaftor-ivacaftor induces rapid clinical improvement in patients with cystic fibrosis (CF) and advanced pulmonary disease, often leading to suspend the indication for lung transplantation. Yet no long-term
Montcouquiol, Sylvie, Murris-Espin, Marlène, Ramel, Sophie, Douvry, Benoit, Martin, Clémence, Lemonnier, Lydie, Burgel, Pierre-Régis, Grenet, Dominique, Hamidfar, Rebecca, da Silva, Jennifer, Durieu, Isabelle, Danner-Boucher, Isabelle, Chiron, Raphaël, Reynaud-Gaubert, Martine, Burnet, Espérie, Paillasseur, Jean-Louis, Leroy, Sylvie, Mely, Laurent   +17 more
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Efficacy and safety of elexacaftor plus tezacaftor plus ivacaftor versus tezacaftor plus ivacaftor in people with cystic fibrosis homozygous for F508del-CFTR: a 24-week, multicentre, randomised, double-blind, active-controlled, phase 3b trial

, 2022
Background: Elexacaftor plus tezacaftor plus ivacaftor is a triple-combination cystic fibrosis transmembrane conductance regulator (CFTR) modulator regimen shown to be generally safe and efficacious in people with cystic fibrosis aged 12 years or older ...
Moskowitz, Samuel M., Ahluwalia, Neil, Peckham, Daniel, Lee, Timothy, Lorenz, Michael, Hilliard, Tom, Vanderhelst, Eef, Duckers, Jamie, Verhulst, Stijn, Van Braeckel, Eva, McKone, Edward F., Bruinsma, Bote G., Thursfield, Rebecca, van Koningsbruggen-Rietschel, Silke, Harris, Christopher, Gleiber, Wolfgang, Stehling, Florian, Vermeulen, Francois, Lou, Yiyue, Downey, Damian G., Aurora, Paul, Wark, Peter, Fischer, Rainald, Nash, Edward F., Mall, Marcus A., Gray, Robert, Tullis, Elizabeth, Lam, Anna P., Roehmel, Jobst, Haworth, Charles, Withers, Nicholas John, Tian, Simon, Barry, Peter, Watson, Danie, Robinson, Philip J., Yuan, Jason, Legg, Julian, Waltz, David, Sutharsan, Sivagurunathan, Mulrennan, Siobhain A., Clements, Barry S., Naehrig, Susanne, Wainwright, Claire E., Doe, Simon James, Smith, Daniel J., MacGregor, Gordon, Barr, Helen Louise   +46 more
core   +4 more sources

The rescue of F508del-CFTR by elexacaftor/tezacaftor/ivacaftor (Trikafta) in human airway epithelial cells is underestimated due to the presence of ivacaftor

, 2021
International audienceTrikafta, currently the leading therapeutic in Cystic Fibrosis (CF), has demonstrated a real clinical benefit. This treatment is the triple combination therapy of two folding correctors elexacaftor/tezacaftor (VX445/VX661) plus the ...
Mirval, Sandra, Carrez, Thomas, Coraux, Christelle, Lévêque, Manuella, Cantereau, Anne, Sage, Edouard, Billet, Arnaud, Becq, Frédéric   +7 more
core   +1 more source

Short-Term Effects of Elexacaftor/Tezacaftor/Ivacaftor Combination on Glucose Tolerance in Young People With Cystic Fibrosis—An Observational Pilot Study [PDF]

, 2022
BACKGROUND: The effect of elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) on glucose tolerance and/or cystic-fibrosis-related diabetes (CFRD) is not well understood.
Kieninger, Elisabeth, Krueger, Linn, Flück, Christa E., Casaulta, Carmen, Latzin, Philipp, Boettcher, Claudia, Bullo, Marina, Flück, Christa E, Korten, Insa   +8 more
core   +1 more source

Personalized Selection of a CFTR Modulator for a Patient with a Complex Allele [L467F;F508del]

Current Issues in Molecular Biology, 2022
The presence of complex alleles in the CFTR gene can lead to difficulties in diagnosing cystic fibrosis and cause resistance to therapy with CFTR modulators.
Elena Kondratyeva, Nataliya Bulatenko, Yuliya Melyanovskaya, Anna Efremova, Elena Zhekaite, Viktoriya Sherman, Anna Voronkova, Irina Asherova, Alexander Polyakov, Tagui Adyan, Valeriia Kovalskaia, Tatiana Bukharova, Dmitry Goldshtein, Sergey Kutsev   +13 more
doaj   +1 more source

Rescue of multiple class II CFTR mutations by elexacaftor+ tezacaftor+ivacaftor mediated in part by the dual activities of Elexacaftor as both corrector and potentiator

, 2020
Positive results in preclinical studies of the triple combination of elexacaftor, tezacaftor and ivacaftor, performed in airway epithelial cell cultures obtained from patients harboring F508del-CFTR, translated to impressive clinical outcomes for ...
Moraes, Theo J, Ouyang, Hong, Gunawardena, Tarini N A, Laselva, Onofrio, Bartlett, Claire, Gonska, Tanja, Bear, Christine E, Eckford, Paul D W   +7 more
core   +1 more source

DataSheet_1_Regulatory T cell enhancement in adults with cystic fibrosis receiving Elexacaftor/Tezacaftor/Ivacaftor therapy.docx

, 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.
Matthias Welsner (11111694), Christian Taube (35629), Dirk Westhölter (14632553), Svenja Straßburg (11111691), Sivagurunathan Sutharsan (3352931), Sebastian Reuter (347960), Jonas Raspe (14494610), Hendrik Uebner (12886982), Mona Schmitz (14632559), Johannes Pipping (14632556)   +9 more
core   +1 more source

Malassezia Folliculitis following Triple Therapy for Cystic Fibrosis

Medicina, 2022
Triple-combination therapy with elexacaftor, tezacaftor and ivacaftor has been recently approved for cystic fibrosis patients with at least one F508del mutation in the transmembrane conductance regulator of the cystic fibrosis gene.
Federica Li Pomi, Luca Di Bartolomeo, Mario Vaccaro, Maria Lentini, Simona Cristadoro, Maria Cristina Lucanto, Mariangela Lombardo, Stefano Costa, Francesco Borgia   +8 more
doaj   +1 more source

Use of elexacaftor/tezacaftor/ivacaftor combination in pregnancy

, 2023
Introduction Management of cystic fibrosis has recently stepped forward with the introduction of cystic fibrosis transmembrane conductance regulator (CFTR) modulators, although data on potential adverse effects are lacking for many categories of patients,
Galoppi, Paola, Brunelli, Roberto, Ascenzioni, Fiorentina, Cimino, Giuseppe, Murciano, Manuel, Botta, Bruno, Sorrenti, Sara   +6 more
core   +1 more source