Results 11 to 20 of about 2,435 (141)
Physiologically Based Pharmacokinetic Modeling of Elexacaftor/Tezacaftor/Ivacaftor in Infants With Cystic Fibrosis [PDF]
CPT: Pharmacometrics & Systems PharmacologyIvacaftor is the only cystic fibrosis transmembrane conductance regulator modulator approved for infants ≥ 1 month. The elexacaftor/tezacaftor/ivacaftor combination, approved for children aged ≥ 2 years, has been shown to significantly slow CF ...
Ngoc Hoa Truong +16 more
doaj +2 more sources
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
Francesca Mattioli +2 more
exaly +3 more sources
Post-approval studies with the CFTR modulators Elexacaftor-Tezacaftor—Ivacaftor
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, Tümmler Burkhard
exaly +3 more sources
Pharmaceuticals, 2022 The use of modulator drugs that target the Cystic Fibrosis transmembrane conductance regulator (CFTR) is the final frontier in the treatment of Cystic Fibrosis (CF), a genetic multiorgan disease.
Dafne Bongiorno +2 more
exaly +3 more sources
Long-term impact of Elexacaftor/Tezacaftor/ivacaftor on pulmonary, nutritional and metabolic outcomes in homozygous F508del cystic fibrosis patients: A real-world cohort study. [PDF]
Br J Clin PharmacolAim Elexacaftor/tezacaftor/ivacaftor (ETI) has markedly improved cystic fibrosis (CF) outcomes. However, its long‐term impact on nutrition, metabolism and liver health remains underexplored. We assessed 30‐month changes in pulmonary, nutritional, metabolic and inflammatory markers in people with CF (PwCF) homozygous for F508del.
Perrotta N +5 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 +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
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 +3 more
doaj +1 more source
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 +2 more
doaj +1 more source
CFTR Modulators in People with Cystic Fibrosis: Real-World Evidence in France
Cells, 2022 Cystic fibrosis (CF) is a rare genetic multisystemic disease, the manifestations of which are due to mutations in the gene encoding the CF transmembrane conductance regulator (CFTR) protein and can lead to respiratory insufficiency and premature death ...
Lucile Regard +4 more
doaj +1 more source