Results 11 to 20 of about 3,864 (165)

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   +3 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

Safety and efficacy of vanzacaftor-tezacaftor-deutivacaftor in adults with cystic fibrosis: Randomised, double-blind, controlled, phase 2 trials [PDF]

, 2023
BACKGROUND: Elexacaftor-tezacaftor-ivacaftor has been shown to be safe and efficacious in people with cystic fibrosis and at least one F508del allele. Our aim was to identify a novel cystic fibrosis transmembrane conductance regulator (CFTR) modulator ...
et al.,, Rubenstein, Ronald C, Uluer, Ahmet Z   +2 more
core   +2 more sources

Standards for the care of people with cystic fibrosis; establishing and maintaining health [PDF]

, 2023
Cystic fibrosis; Health; StandardsFibrosis quística; Salud; EstándaresFibrosi quística; Salut; EstàndardsThis is the second in a series of four papers updating the European Cystic Fibrosis Society (ECFS) standards for the care of people with CF.
Addy, Charlotte, Bell, Scott C, Bevan, Amanda, Borawska, Urzula, Brown, Catherine, Burgel, Pierre-Régis, Button, Brenda, Castellani, Carlo, Chansard, Audrey, Chilvers, Mark, Davies, Gwyneth, Davies, Jane C, De Boeck, Kris, Declercq, Dimitri, Doumit, Michael, Drevinek, Pavel, et al, Fajac, Isabelle, Gartner, Silvia, Georgiopoulos, Anna M, Gramegna, Andrea, Gursli, Sandra, Hansen, Carina M E, Hug, Martin J, Lammertyn, Elise, Landau, Edwina (Eddie) C, Langley, Ross, Mayer-Hamblett, Nicole, Middleton, Anna, Middleton, Peter G, Radtke, Thomas, Southern, Kevin W   +31 more
core   +7 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

Worldwide rates of diagnosis and effective treatment for cystic fibrosis [PDF]

, 2022
BACKGROUND: Time has seen management for Cystic Fibrosis (CF) advance drastically, most recently in the development of the disease-modifying triple combination therapy ivacaftor/tezacaftor/elexacaftor.
Garratt, A, Guo, J, Hill, A
core   +1 more source

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

Extracellular phosphate enhances the function of F508del-CFTR rescued by CFTR correctors [PDF]

, 2021
Background: The clinical response to cystic fibrosis transmembrane conductance regulator (CFTR) modulators varies between people with cystic fibrosis (CF) of the same genotype, in part through the action of solute carriers encoded by modifier genes. Here,
Delpiano, Livia, Gray, Michael A, Lin, JinHeng, Saint-Criq, Vinciane, Sheppard, David N, Wang, Yiting   +5 more
core   +3 more sources

Cystic fibrosis year in review 2022 [PDF]

, 2023
Remarkable medical advancements have been made for people with cystic fibrosis (CF) in recent years, with an abundance of research continuing to be conducted worldwide.
Kim, Jeeyeon, Lyman, Benjamin, Savant, Adrienne P.   +2 more
core   +2 more sources

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