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The Lancet. Respiratory Medicine|September 6, 2025
Sweat chloride and lung function responses to elexacaftor-tezacaftor-ivacaftor in people with cystic fibrosis with two versus one responsive CFTR variants: an analysis of two real-world observational studiesPierre-Régis Burgel, Jennifer Da Silva, Emmanuelle Girodon, et al.ERJ Open Research|June 18, 2026
Persistence of lung hyperinflation and small airway dysfunction in school-aged children with cystic fibrosis treated with elexacaftor-tezacaftor-ivacaftor: results from the real-world MODUL-CF studyLucie Colin, Véronique Delaup, Muriel Le Bourgeois, et al.Clinical Pharmacokinetics|February 4, 2024
Lumacaftor/Ivacaftor Population Pharmacokinetics in Pediatric Patients with Cystic Fibrosis: A First Step Toward Personalized TherapyNaïm Bouazza, Saïk Urien, Frantz Foissac, et al.American Journal of Respiratory and Critical Care Medicine|July 11, 2022
Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Children 6 Through 11 Years of Age with Cystic Fibrosis Heterozygous for <i>F508del</i> and a Minimal Function Mutation: A Phase 3b, Randomized, Placebo-controlled StudyMarcus A Mall, Rossa Brugha, Silvia Gartner, et al.Pediatric Pulmonology|March 15, 2019
Health-related quality of life in infants and children with interstitial lung diseaseClara Lauby, Pierre-Yves Boelle, Rola Abou Taam, et al.Clinical and Translational Science|May 10, 2025
Elexacaftor/Tezacaftor/Ivacaftor Population Pharmacokinetics in Pediatric Patients With Cystic FibrosisNgoc Hoa Truong, Sihem Benaboud, Naïm Bouazza, et al.The Lancet. Respiratory Medicine|August 16, 2024
The expanded French compassionate programme for elexacaftor-tezacaftor-ivacaftor use in people with cystic fibrosis without a F508del CFTR variant: a real-world studyPierre-Régis Burgel, Isabelle Sermet-Gaudelus, Emmanuelle Girodon, et al.Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society|March 25, 2025
First real-world study of fetal therapy with CFTR modulators in cystic fibrosis: Report from the MODUL-CF studyAnne-Sophie Bonnel, Tiphaine Bihouée, Mélanie Ribault, et al.The Journal of Clinical Endocrinology and Metabolism|February 20, 2025
Impact of elexacaftor/tezacaftor/ivacaftor on glucose tolerance in adolescents with cystic fibrosisAlfonso Galderisi, Laurence Weiss, Alix Besançon, et al.Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society|July 8, 2023
Nonsense mutations accelerate lung disease and decrease survival of cystic fibrosis childrenAnnalisa Orenti, Iwona Pranke, Caroline Faucon, et al.Pageof 12