Results 11 to 20 of about 6,598 (226)

Impact of Elexacaftor-Tezacaftor-Ivacaftor on Quality of Life in Children With Cystic Fibrosis. [PDF]

Pediatr Pulmonol
ABSTRACT Objectives CFTR modulators have revolutionized cystic fibrosis (CF) management by targeting the defective protein rather than its consequences. Their impact on quality of life (QoL) have been studied in numerous trials, but few data are available on QoL in patients receiving Elexacaftor‐Tezacaftor‐Ivacaftor (ETI), notably in children given its
Kümmerli S, Elviro CF, Regamey N, Mornand A, Faouzi M, Rochat I, Blanchon S.   +6 more
europepmc   +2 more sources

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

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

Real-life impact of highly effective CFTR modulator therapy in children with cystic fibrosis

Frontiers in Pharmacology, 2023
Introduction: Recently, cystic fibrosis transmembrane regulator modulator therapy with elexacaftor/tezacaftor/ivacaftor has become available for children with cystic fibrosis (CF) carrying at least one F508del mutation.Objective: To assess the ...
Margarete Olivier, Alexandra Kavvalou, Matthias Welsner, Raphael Hirtz, Svenja Straßburg, Sivagurunathan Sutharsan, Florian Stehling, Mathis Steindor   +7 more
doaj   +1 more source

Cystic fibrosis modulator therapy can reverse cystic bronchiectasis

Respirology Case Reports, 2023
Bronchiectasis is often considered progressive and irreversible, so cases of regression or reversal are an important step in understanding the underlying pathophysiological mechanisms.
Peter G. Middleton, Nicholas J. Simmonds
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

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

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

Combined CFTR modulator therapies are linked with anabolic benefits and insulin-sparing in cystic fibrosis-related diabetes

Journal of Clinical & Translational Endocrinology, 2023
Aims: Combined CFTR modulator therapies have dramatically altered pulmonary outcomes in patients with cystic fibrosis (CF). Their impact on glucose metabolism requires further investigations.
Fabian Lurquin, Sophie Gohy, Michel P. Hermans, Vanessa Preumont   +3 more
doaj   +1 more source

Evaluation of Elexacaftor/Tezacaftor/Ivacaftor-Mediated Drug-Induced Liver Injury Using a Liver-On-Chip Model. [PDF]

Clin Transl Sci
ABSTRACT Elexacaftor/tezacaftor/ivacaftor (ETI), a cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapy, has provided great improvements in lung function and well‐being for people with CF. The use of ETI has been complicated by reports of rare but significant liver function test elevations in clinical trials and drug‐induced ...
Shi A, Agarwal P, Liu ZX, Beringer PM.
europepmc   +2 more sources