Results 21 to 30 of about 3,564 (173)

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

Efficacy and Safety of Elexacaftor-Tezacaftor-Ivacaftor in the Treatment of Cystic Fibrosis: A Systematic Review

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, Maria Moustaki, Konstantinos Douros, Ioanna Loukou   +3 more
doaj   +1 more source

Efficacy and safety of elexacaftor plus tezacaftor plus ivacaftor versus tezacaftor plus ivacaftor in people with cystic fibrosis homozygous for F508del-CFTR: a 24-week, multicentre, randomised, double-blind, active-controlled, phase 3b trial

, 2021
BackgroundElexacaftor plus tezacaftor plus ivacaftor is a triple-combination cystic fibrosis transmembrane conductance regulator (CFTR) modulator regimen shown to be generally safe and efficacious in people with cystic fibrosis aged 12 years or older ...
Siobhain A Mulrennan, Damian Downey, Watson, D., MacGregor, G., Verhulst, S., David Waltz, Timothy Lee, McKone, E.F., Downey, D., Haworth, C., Rainald Fischer, Wainwright, C.E., Doe, S.J., Barry, P., Eef Vanderhelst, Peter Wark, Stehling, F., Elizabeth Tullis, Yiyue Lou, Jamie Duckers, Sivagurunathan Sutharsan, Julian Legg, Florian Stehling, Downey, D.G., Bruinsma, B.G., Tom Hilliard, Smith, D.J., Nicholas John Withers, Danie Watson, Helen Louise Barr, Naehrig, S., Ahluwalia, N., Aurora, P., Gordon MacGregor, Gleiber, W., Yuan, J., Neil Ahluwalia, Hilliard, T., Duckers, J., Gray, R., Daniel J Smith, Michael Lorenz, Silke van Koningsbruggen-Rietschel, Barry S Clements, Jobst Roehmel, Stijn Verhulst, Thursfield, R., Wark, P., Eva Van Braeckel, Lam, A.P., Simon Tian, Francois Vermeulen, Nash, E.F., Edward F McKone, Harris, C., Robinson, P.J., Damian G Downey, Peckham, D., Robert Gray, Lorenz, M., Tian, S., Vermeulen, F., Jason Yuan, Marcus A Mall, Paul Aurora, Lou, Y., Wolfgang Gleiber, Waltz, D., Anna P Lam, Mall, M.A., Roehmel, J., van Koningsbruggen-Rietschel, S., Claire E Wainwright, Tullis, E., Susanne Naehrig, Legg, J., Clements, B.S., Sutharsan, S., Van Braeckel, E., Fischer, R., Vanderhelst, E., Christopher Harris, Simon James Doe, Lee, T., Mulrennan, S.A., Edward F Nash, Bote G Bruinsma, Charles Haworth, Withers, N.J., Rebecca Thursfield, Daniel Peckham, Samuel M Moskowitz, Barr, H.L., Philip J Robinson, Peter Barry, Moskowitz, S.M.   +95 more
core   +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 Pharmacol
Aim 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, Fiorito LA, Vescovo R, Virgilio A, Amato G, Cimino G.   +5 more
europepmc   +2 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, Burkhard Tümmler
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, Clémence Martin, Espérie Burnet, Jennifer Da Silva, Pierre-Régis Burgel   +4 more
doaj   +1 more source

Simultaneous Quantification of Ivacaftor, Tezacaftor, and Elexacaftor in Cystic Fibrosis Patients’ Plasma by a Novel LC–MS/MS Method

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
Federica Pigliasco, Alessia Cafaro, Manuela Stella, Giammarco Baiardi, Sebastiano Barco, Nicoletta Pedemonte, Claudia D’Orsi, Federico Cresta, Rosaria Casciaro, Carlo Castellani, Maria Grazia Calevo, Francesca Mattioli, Giuliana Cangemi   +12 more
doaj   +1 more source

DataSheet1_Changes in cystic fibrosis transmembrane conductance regulator protein expression prior to and during elexacaftor-tezacaftor-ivacaftor therapy.PDF

, 2023
Background: Defects in expression, maturation or function of the epithelial membrane glycoprotein CFTR are causative for the progressive disease cystic fibrosis.
Frauke Stanke (32239), Gesine Hansen (194515), Burkhard Tümmler (4273), Rebecca Minso (14512229), Sibylle Junge (494286), Anna-Maria Dittrich (306619), Ella M. Eichhorn (14512226), Silke Hedtfeld (14512223), Sophia T. Pallenberg (14512220), Annette Sauer-Heilborn (691740), Stephanie Tamm (117858), Tobias Welte (87188), Felix C. Ringshausen (14512232)   +12 more
core   +1 more source

Gene Therapy: A Possible Alternative to CFTR Modulators?

Frontiers in Pharmacology, 2021
Cystic fibrosis (CF) is a rare genetic disease that affects several organs, but lung disease is the major cause of morbidity and mortality. The gene responsible for CF, the CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) gene, has been ...
J. Mercier, M. Ruffin, H. Corvol, H. Corvol, L. Guillot   +4 more
doaj   +1 more source

Colorectal Cancer Complicated by Pulmonary Embolism and Potential Takotsubo Cardiomyopathy in a Person With Cystic Fibrosis: Diagnostic and Management Considerations. [PDF]

Respirol Case Rep
We describe a complex case of pulmonary embolism and dilated cardiomyopathy in a person with cystic fibrosis. These late complications occurred following laparoscopic left hemicolectomy for colonic adenocarcinoma. This case highlights the complexities of evaluating breathlessness in severe lung disease, and the management challenges that arise as life ...
Frederiksen L, Brown J, Moore V, Reid DW.   +3 more
europepmc   +2 more sources