Results 1 to 10 of about 2,230 (120)

Rethinking hyperbilirubinemia: Gilbert syndrome in children with cystic fibrosis, a case report [PDF]

Respiratory Medicine Case Reports
Cystic Fibrosis leads to liver complications, including cystic fibrosis liver disease but hyperbilirubinemia in CF patients on CFTR modulators is less understood.
Yara Salameh, John Lyles, Shatha Yousef
doaj   +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

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

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

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

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

Elexacaftor-Tezacaftor-Ivacaftor Treatment Reduces Abdominal Symptoms in Cystic Fibrosis-Early results Obtained With the CF-Specific CFAbd-Score

Frontiers in Pharmacology, 2022
Background: The novel and highly effective CFTR modulator combination of elexacaftor-tezacaftor-ivacaftor (ETI) has been shown to improve lung function and body weight in people with Cystic Fibrosis (pwCF) carrying a F508del mutation. However, the impact
Jochen G. Mainz, Jochen G. Mainz, Carlos Zagoya, Louise Polte, Lutz Naehrlich, Lutz Naehrlich, Lenny Sasse, Olaf Eickmeier, Christina Smaczny, Anton Barucha, Lilith Bechinger, Franziska Duckstein, Ludwik Kurzidim, Ludwik Kurzidim, Patience Eschenhagen, Laura Caley, Daniel Peckham, Daniel Peckham, Carsten Schwarz   +18 more
doaj   +1 more source

Elexacaftor is a CFTR potentiator and acts synergistically with ivacaftor during acute and chronic treatment

Scientific Reports, 2021
Cystic fibrosis (CF) is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR), which lead to early death due to progressive lung disease. The development of small-molecule modulators that directly interact with CFTR to aid
Ciaran A. Shaughnessy, Pamela L. Zeitlin, Preston E. Bratcher   +2 more
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

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