Results 151 to 160 of about 4,690 (200)

Patient perspectives on elexacaftor/tezacaftor/ivacaftor after lung transplant

Journal of Cystic Fibrosis
Cystic fibrosis transmembrane conductance regulator (CFTR) modulators, including elexacaftor/tezacaftor/ivacaftor (ETI), significantly improve outcomes and quality of life for people with cystic fibrosis (CF). However, little is known about how lung transplant recipients (LTRs) perceive the use of ETI.
Dave Young, Lauren E. Bartlett, Jennifer Guimbellot, Tijana Milinic, Nora Burdis, Eliana R. Gill, Erika D. Lease, Christopher H. Goss, Siddhartha G. Kapnadak, Kathleen J. Ramos   +9 more
openaire   +2 more sources

Long-Term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Adults and Adolescents with Cystic Fibrosis and at Least One F508del Allele: A Phase 3, Open-Label Extension Study.

American Journal of Respiratory and Critical Care Medicine
RATIONALE Clinical and real-world studies show elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) is efficacious and safe in people with cystic fibrosis (CF) ≥12 years of age with at least one F508del allele.
C. Daines, Deepika Polineni, Elizabeth Tullis, Stefano Costa, Rachel W. Linnemann, M. Mall, E. McKone, Bradley S. Quon, F. Ringshausen, H. Selvadurai, J. Taylor-Cousar, Nicholas J. Withers, Gregory S. Sawicki, Tim W. R. Lee, N. Ahluwalia, Jessica Morlando Geiger, Mark Jennings, Yaoyuan Vincent Tan, David Waltz, Bonnie W Ramsey, Matthias Griese   +20 more
semanticscholar   +1 more source

Elexacaftor/tezacaftor/ivacaftor outpatient desensitization

Annals of Allergy, Asthma & Immunology, 2022
Ramya Balijepally, Denise Kwong, Linda Zhu, Juanita Valdes Camacho, Anne Liu   +4 more
openaire   +2 more sources

Evaluation of the response to elexacaftor-tezacaftor-ivacaftor of the rare CFTR variants L383S, I507del, L1065P and R1066H in intestinal organoid-derived epithelial monolayers.

Journal of Cystic Fibrosis
INTRODUCTION Cystic fibrosis (CF) is caused by mutation of the CFTR gene, encoding an epithelial anion channel. Here we evaluated the effect of the modulator combination elexacaftor-tezacaftor-ivacaftor (ETI) on the function of four rare, poorly ...
Jessica Conti, Dora Angyal, K. Kleinfelder, R. V. Latorre, Martina Calicchia, A. Farinazzo, Luca Rodella, F. Tomba, A. Massella, Luca Frulloni, G. Taccetti, V. Terlizzi, Anny Leung, T. Groeneweg, M. Bijvelds, P. Melotti, Claudio Sorio   +16 more
semanticscholar   +1 more source

Nasal Epithelium Transcriptomics Predict Clinical Response to Elexacaftor/Tezacaftor/Ivacaftor

American Journal of Respiratory Cell and Molecular Biology
Elexacaftor/tezacaftor/ivacaftor (ETI) has had a substantial positive impact for people living with cystic fibrosis (pwCF). However, there can be substantial variability in efficacy, and we lack adequate biomarkers to predict individual response. We thus aimed to identify transcriptomic profiles in nasal respiratory epithelium that predict clinical ...
Molin Yue, Daniel J. Weiner, Kristina M. Gaietto, Franziska J. Rosser, Christopher M. Qoyawayma, Michelle L. Manni, Michael M. Myerburg, Joseph M. Pilewski, Juan C. Celedón, Wei Chen, Erick Forno   +10 more
openaire   +2 more sources

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. M. Renz, Frank Wacker, Anna-Maria Dittrich, Jens Vogel-Claussen   +11 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

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, Jean 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, Raphael Chiron, L. Weiss, I. Fajac, J. da Silva, Clémence Martin   +21 more
semanticscholar   +1 more source

Real-world outcomes of generic elexacaftor/tezacaftor/ivacaftor (gETI) in South Africans (SA) with CF using standard versus clarithromycin-boosted gETI, modulator-sparing strategies to reduce cost.

Journal of Cystic Fibrosis
OBJECTIVE Access to highly effective modulator therapies (HEMT) in resource-limited countries is limited by prohibitive cost and restrictive patents.
M. Zampoli, J. Verstraete, C. Baird, Tony Biebuyck, G. Calligaro, M. Coetzee, C. Els, M. Frauendorf, P. Gebers, B. Morrow, D. Richards, H. Truter, A. Hill   +12 more
semanticscholar   +1 more source