Results 151 to 160 of about 9,288 (216)

The French compassionate programme of elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis with advanced lung disease and no F508del CFTR variant

European Respiratory Journal, 2023
Background The European Medicines Agency has approved the cystic fibrosis transmembrane conductance regulator (CFTR) modulator combination elexacaftor/tezacaftor/ivacaftor (ETI) for people with cystic fibrosis (CF) carrying at least one F508del variant ...
P. Burgel, I. Sermet-Gaudelus, I. Durieu, R. Kanaan, J. Macey, D. Grenet, M. Porzio, N. Coolen-Allou, R. Chiron, C. Marguet, B. Douvry, N. Dufeu, I. Danner-Boucher, P. Foucaud, L. Lemonnier, E. Girodon, J. da Silva, Clémence Martin, Claire Pascaline Bénédicte Julie Sylvie Benoit Natacha An Andrejak Priou Richaud-Thiriez Macey Montcouquiol   +18 more
semanticscholar   +1 more source

The expanded French compassionate programme for elexacaftor-tezacaftor-ivacaftor use in people with cystic fibrosis without a F508del CFTR variant: a real-world study.

The Lancet Respiratory Medicine
BACKGROUND Elexacaftor-tezacaftor-ivacaftor has been approved in Europe for people with cystic fibrosis with at least one F508del CFTR variant. Additionally, it is approved by the US Food and Drug Administration (FDA) for people with cystic fibrosis with
Pierre-Régis Burgel, I. Sermet-Gaudelus, E. Girodon, Isabelle Durieu, V. Houdouin, Camille Audousset, J. Macey, Dominique Grenet, Michele Porzio, Marlène Murris-Espin, P. Reix, Mélisande Baravalle, Chantal Belleguic, Laurent Mely, Juliette Verhille, Laurence Weiss, Martine Reynaud-Gaubert, M. Mittaine, R. Hamidfar, Sophie Ramel, L. Cosson, B. Douvry, Isabelle Danner-Boucher, P. Foucaud, C. Roy, Espérie Burnet, C. Raynal, M. Audrézet, J. da Silva, Clémence Martin   +29 more
semanticscholar   +1 more source

Reduction of systemic inflammation by elexacaftor/tezacaftor/ivacaftor correlates with lung function improvement in cystic fibrosis

European Respiratory Journal
Background The triple cystic fibrosis transmembrane conductance regulator modulator therapy elexacaftor/tezacaftor/ivacaftor (ETI) rapidly improves airway and systemic inflammation in people with cystic fibrosis. However, longitudinal effects on systemic
O. Halle, S. Graeber, J. Kontsendorn, C. Kessemeier, Jan-Niklas Falke, J. Schwabe, K. Schütz, S. Pallenberg, R. Dalferth, R. Grychtol, Felix C. Ringshausen, M. Stahl, Stephanie Thee, Jobst F Roehmel, Z. Syunyaeva, J. Duerr, Jaehi Chung, S. Hirtz, T. Uselmann, I. Kühbandner, C. Rückes-Nilges, A. Bagheri-Pothoff, S. Barth, B. Schaub, F. Brinkmann, S. Weber, S. van Koningsbruggen-Rietschel, Mustafa Abdo, Markus Weckmann, Stefanie Widder, G. Hansen, B. Tümmler, O. Sommerburg, L. Naehrlich, M. Mall, Anna-Maria Dittrich   +35 more
semanticscholar   +1 more source

Multisystemic Effects of Elexacaftor-Tezacaftor-Ivacaftor in Adults with Cystic Fibrosis and Advanced Lung Disease.

Annals of the American Thoracic Society
RATIONALE Limited data exist on safety and effectiveness of elexacaftor-tezacaftor-ivacaftor (ETI) in people with cystic fibrosis (pwCF) and advanced lung disease.
Pierre-Régis Burgel, J. Paillasseur, Isabelle Durieu, Martine Reynaud-Gaubert, R. Hamidfar, Marlène Murris-Espin, Isabelle Danner-Boucher, R. Chiron, Sylvie Leroy, B. Douvry, Dominique Grenet, Laurent Mely, Sophie Ramel, Sylvie Moncouquiol, Espérie Burnet, El Hassane Ouaalaya, Philippe Sogni, J. da Silva, Clémence Martin   +18 more
semanticscholar   +1 more source

Elexacaftor/Tezacaftor/Ivacaftor Effect on Bone Density and Body Composition: A Retrospective Analysis

Pediatric Pulmonology
The approval of cystic fibrosis transmembrane conductance regulator modulators elexacaftor/tezacaftor/ivacaftor (ETI), has significantly improved pulmonary function for people with cystic fibrosis (pwCF).
S. Trost, T. Harindhanavudhi, Qi Wang, A. Ankireddypalli, Azmi Simrah, Sreekanth Avula, Amir Moheet   +6 more
semanticscholar   +1 more source

PREFUL MRI for Monitoring Perfusion and Ventilation Changes after Elexacaftor-Tezacaftor-Ivacaftor Therapy for Cystic Fibrosis: A Feasibility Study.

Radiology: Cardiothoracic Imaging
Purpose To assess the feasibility of monitoring the effects of elexacaftor-tezacaftor-ivacaftor (ETI) therapy on lung ventilation and perfusion in people with cystic fibrosis (CF), using phase-resolved functional lung (PREFUL) MRI.
Martha Dohna, A. Voskrebenzev, F. Klimeš, Till F. Kaireit, J. Glandorf, S. Pallenberg, Felix C. Ringshausen, G. Hansen, D. Renz, Frank Wacker, Anna-Maria Dittrich, J. Vogel-Claussen   +11 more
semanticscholar   +1 more source

Outcomes of prenatal use of elexacaftor/tezacaftor/ivacaftor in carrier mothers to treat meconium ileus in fetuses with cystic fibrosis.

Journal of Cystic Fibrosis
As cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies including elexacaftor/tezacaftor/ivacaftor (ETI) have become widely used in eligible patients with cystic fibrosis (CF), the use of these medications in pregnant people has
A. Metcalf, S. Martiniano, S. Sagel, Michael V. Zaretsky, E. Zemanick, J. Hoppe   +5 more
semanticscholar   +1 more source

Gathering real-world compassionate data to expand eligibility for elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis with N1303K or other rare CFTR variants: a viewpoint

European Respiratory Journal
Extract Elexacaftor/tezacaftor/ivacaftor (ETI) was approved in 2019 by the US Food and Drug Administration (FDA) and in 2020 by the European Medicines Agency (EMA) for people with cystic fibrosis (CF).
Pierre-Régis Burgel, I. Sermet-Gaudelus, E. Girodon, R. Kanaan, J. Le Bihan, Natascha Remus, B. Ravoninjatovo, Dominique Grenet, Michele Porzio, V. Houdouin, Laurence Le Clainche-Viala, Isabelle Durieu, R. Nove-Josserand, Jeanne Languepin, B. Coltey, A. Guillaumot, Camille Audousset, R. Chiron, L. Weiss, I. Fajac, J. da Silva, Clémence Martin   +21 more
semanticscholar   +1 more source

Drug-induced liver injury associated with elexacaftor/tezacaftor/ivacaftor: A pharmacovigilance analysis of the FDA adverse event reporting system (FAERS).

Journal of Cystic Fibrosis
INTRODUCTION The efficacy and safety of elexacaftor/tezacaftor/ivacaftor (ETI) have been established in prospective clinical trials. Liver function test elevations were observed in a greater proportion of patients receiving ETI compared with placebo ...
Alan Shi, Harold Nguyen, C. Kuo, P. Beringer   +3 more
semanticscholar   +1 more source

Elexacaftor-Tezacaftor-Ivacaftor and Ivacaftor (Trikafta)

Canadian Journal of Health Technologies
Reimbursement reviews are comprehensive assessments of the clinical effectiveness and cost-effectiveness, as well as patient and clinician perspectives, of a drug or drug class. The assessments inform nonbinding recommendations that help guide the reimbursement decisions of Canada’s federal, provincial, and territorial governments, with the ...
openaire   +1 more source