Results 11 to 20 of about 4,057 (184)

Post-approval studies with the CFTR modulators Elexacaftor-Tezacaftor—Ivacaftor [PDF]

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   +3 more sources

Successful pregnancy in a cystic fibrosis patient with a severe impairment of lung function receiving Elexacaftor-Tezacaftor-Ivacaftor [PDF]

Respiratory Medicine Case Reports, 2022
Before the arrival of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) modulators women with CF and impaired lung function were experiencing a high risk of complications and mortality during and the years after pregnancy.
Zisis Balmpouzis, Annabelle Faure van Rossum, David Baud, Alice Panchaud, Georgia Mitropoulou, Jesica Mazza Stalder, Angela Koutsokera   +6 more
doaj   +5 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

Altered functional interactions between CFTR disease mutants ΔF508 and G551D and the protein kinase A catalytic subunit. [PDF]

J Physiol
Abstract figure legend Pathogenic mutations ΔF508 and G551D of the cystic fibrosis transmembrane conductance regulator (CFTR) channel alter its interactions with protein kinase A (PKA). Top left, for both mutants, but not for wild‐type (WT) CFTR, non‐catalytic stimulation of channel activity by PKA is larger in the presence of N6‐(2‐phenylethyl)‐ATP (P‐
Závoti O, Simon MA, Csanády L.
europepmc   +2 more sources

Standards for the care of people with cystic fibrosis; establishing and maintaining health [PDF]

, 2023
Cystic fibrosis; Health; StandardsFibrosis quística; Salud; EstándaresFibrosi quística; Salut; EstàndardsThis is the second in a series of four papers updating the European Cystic Fibrosis Society (ECFS) standards for the care of people with CF.
Addy, Charlotte, Bell, Scott, Bevan, Amanda, Borawska, Urzula, Brown, Catherine, Gartner Tizzano, Silvia, Southern, Kevin   +6 more
core   +7 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

Impact of COVID-19 and CFTR Modulators on Cystic Fibrosis: A Real-World Analysis of Care Patterns. [PDF]

Pediatr Pulmonol
ABSTRACT Introduction Novel therapeutics and rapid expansion of telehealth have reshaped cystic fibrosis (CF) care; however, the impact on visit patterns and equitable access across the CF population remains unclear. We characterized changes in visit patterns from 2017 to 2022 and the association of sociodemographic and clinical factors with visit ...
Hinton AC, Sears EH, Lopez-Pintado S, Zuckerman JB.   +3 more
europepmc   +2 more sources