Results 1 to 10 of about 3,589 (157)

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   +6 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]

American Journal of Respiratory and Critical Care Medicine, 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 ...
Marcus Mall, Rossa Brugha, Silvia Gartner   +2 more
exaly   +2 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   +5 more sources

Impact of elexacaftor/tezacaftor/ivacaftor on the small airways in cystic fibrosis. [PDF]

J Bras Pneumol
Objective: To evaluate the impact of the elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) combination on the small airways in adults with cystic fibrosis (CF), a genetic disorder that primarily affects the respiratory system, leading to progressive lung ...
Silva SC, Barroso A, Cunha M, Fragoso E, Azevedo P.   +4 more
europepmc   +3 more sources

Scedosporium infection as a late complication after start of elexacaftor/tezacaftor/ivacaftor: two case reports [PDF]

Respiratory Medicine Case Reports
Highly effective modulator treatment (HEMT) has resulted in an improved prognosis for people with cystic fibrosis (pwCF). In this case report, we present two pwCF with clinical stabilization after start of HEMT who developed complicated fungal infections
P van Mourik, MD PhD, E.C. van der Hout, MD, H.D. Luijk, MD PhD, R.W. Hofland, MD PhD, A.H.W. Bruns, MD PhD, H.G.M. Heijerman, MD PhD, I. Bronsveld, MD PhD   +6 more
doaj   +2 more sources

Elexacaftor–tezacaftor–ivacaftor for cystic fibrosis with Phe508del mutation: Evidence from randomized controlled trials

SAGE Open Medicine
Objective: This study aimed to conduct a systematic review and meta-analysis of randomized controlled trials to evaluate the effects of elexacaftor–tezacaftor–ivacaftor (ELX-TEZ-IVA) on patients with cystic fibrosis (CF). Methods: A systematic search was
Fei Lin
exaly   +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

People with cystic fibrosis with high sputum neutrophil elastase on elexacaftor-tezacaftor-ivacaftor exhibit worse pulmonary function and pro-inflammatory airway milieu [PDF]

Scientific Reports
Cystic fibrosis (CF) is a genetic condition affecting over 100,000 individuals worldwide. Lung disease is the main cause of mortality in CF, with chronic neutrophilic inflammation as a hallmark.
Alexandre Cammarata-Mouchtouris, Diego Moncada Giraldo, Genoah L. Collins, Maria M. Parrilla, Susan O. Kim, Sarah Mansour, Vincent D. Giacalone, Kirsten A. Cottrill, Rabindra Tirouvanziam, Joshua D. Chandler   +9 more
doaj   +2 more sources

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   +5 more sources

Elexacaftor/Tezacaftor/Ivacaftor Population Pharmacokinetics in Pediatric Patients With Cystic Fibrosis [PDF]

Clinical and Translational Science
Elexacaftor/tezacaftor/ivacaftor (ETI) significantly improves treatment outcomes for people with cystic fibrosis (pwCF) with at least one F508del allele. In 2023, the Food and Drug Administration approved ETI for children with CF aged 2–5 years. However,
Ngoc Hoa Truong, Sihem Benaboud, Naïm Bouazza, Mahassen Barboura, Emmanuelle Bardin, Michel Miralles, Gabrielle Lui, Léo Froelicher‐Bournaud, Steeve Rouillon, Tiphaine Bihouee, Stéphanie Bui, Philippe Reix, Marie‐Laure Dalphin, Muriel Laurans, Jeanne Languepin, Harriet Corvol, Françoise Troussier, Laurence Weiss, Rames Cinthia, Aurélie Tatopoulos, Eric Deneuville, Raphael Chiron, Nathalie Stremler, Cathy Llerena, Sophie Ramel, Caroline Perisson, Véronique Houdoin, Marie Mittaine, Jean‐Marc Treluyer, Isabelle Sermet‐Gaudelus, Frantz Foissac, MODUL‐CF study group   +31 more
doaj   +2 more sources