Results 1 to 10 of about 4,690 (200)

Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele

New England Journal of Medicine, 2019
BACKGROUND Cystic fibrosis is caused by mutations in the gene encoding the cystic fibrosis transmembrane conductance regulator (CFTR) protein, and nearly 90% of patients have at least one copy of the Phe508del CFTR mutation.
Peter Gordon Middleton, Marcus A Mall, Larry C Lands   +2 more
exaly   +5 more sources

Elexacaftor-Tezacaftor-Ivacaftor: A Life-Changing Triple Combination of CFTR Modulator Drugs for Cystic Fibrosis

Pharmaceuticals, 2023
Cystic fibrosis (CF) is a potentially fatal monogenic disease that causes a progressive multisystemic pathology. Over the last decade, the introduction of CF transmembrane conductance regulator (CFTR) modulator drugs into clinical practice has profoundly
Mafalda Bacalhau, Mariana Camargo, Grace A. V. Magalhães-Ghiotto, Sybelle Drumond, Carlos Henrique M. Castelletti, Miquéias Lopes-Pacheco   +5 more
doaj   +2 more sources

Changes in sputum viscoelastic properties and airway inflammation in primary ciliary dyskinesia are comparable to cystic fibrosis on elexacaftor/tezacaftor/ivacaftor therapy. [PDF]

Eur Respir J
Background Primary ciliary dyskinesia (PCD) and cystic fibrosis (CF) are muco-obstructive lung diseases that are caused by distinct genetically determined defects in mucociliary clearance; however, knowledge on the relative severity of airway mucus ...
Nussstein H, Urbantat RM, Fentker K, Loewe A, Duerr J, Haji M, Doellinger F, Stahl M, Graeber SY, Gradzielski M, Röhmel J, Mertins P, Schaupp L, Mall MA.   +13 more
europepmc   +2 more sources

Effect of elexacaftor/tezacaftor/ivacaftor on systemic inflammation in cystic fibrosis. [PDF]

Thorax
Background Despite significant clinical improvements, there is evidence of persisting airway inflammation in people with cystic fibrosis (CF) established on elexacaftor/tezacaftor/ivacaftor (ETI) therapy.
Maher RE, Cytlak-Chaudhuri UM, Aleem S, Barry P, Brice DP, Caamaño Gutiérrez E, Driver K, Emmott E, Rothwell A, Smith E, Travis M, Lee D, McNamara PS, Waller I, Smith JA, Jones A, Lord RW.   +16 more
europepmc   +2 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

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

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

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

Clinical Effectiveness of Elexacaftor/Tezacaftor/Ivacaftor in People with Cystic Fibrosis: A Clinical Trial

American Journal of Respiratory and Critical Care Medicine, 2021
Rationale The cystic fibrosis (CF) modulator drug, elexacaftor/tezacaftor/ivacaftor (ETI), proved highly effective in controlled clinical trials for individuals with at least one F508del allele, which occurs in at least 85% of people with CF.
D. Nichols, A. Paynter, S. Heltshe, S. Donaldson, C. Frederick, S. Freedman, D. Gelfond, L. Hoffman, A. Kelly, M. Narkewicz, J. Pittman, F. Ratjen, M. Rosenfeld, S. Sagel, S. Schwarzenberg, P. Singh, G. Solomon, M. Stalvey, J. Clancy, S. Kirby, J. V. Van Dalfsen, M. Kloster, S. Rowe   +22 more
semanticscholar   +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