Results 21 to 30 of about 6,985 (222)

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

Heterogeneity of weight gain after initiation of Elexacaftor/Tezacaftor/Ivacaftor in people with cystic fibrosis [PDF]

, 2023
Background: The introduction of the novel therapy, Elexacaftor/Tezacaftor/Ivacaftor (ETI) has been effective in improving weight gain in both clinical trials and real-world studies. However, the magnitude of this effect appears to be heterogeneous across
Aliberti, Stefano, Alicandro, Gianfranco, Amati, Francesco, Blasi, Francesco, Contarini, Martina, Cristiani, Luca, Fiocchi, Alessandro Giovanni, Gramegna, Andrea, Leonardi, Gloria, Majo, Fabio   +9 more
core   +1 more source

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

, 2023
This is the second in a series of four papers updating the European Cystic Fibrosis Society (ECFS) standards for the care of people with CF. This paper focuses on establishing and maintaining health.
Addy, Charlotte, Bell, Scott, Bertrand, Dominique Pougheon, Bevan, Amanda, Borawska, Urzula, Brown, Catherine, Burgel, Pierre-Régis, Button, Brenda, Castellani, Carlo, Chansard, Audrey, Chilvers, Mark, Davies, Gwyneth, Davies, Jane, De Boeck, Kris, Declercq, Dimitri, Doumit, Michael, Drevinek, Pavel, Fajac, Isabelle, Gartner, Silvia, Georgiopoulos, Anna, Gramegna, Andrea, Gursli, Sandra, Hansen, Carina Me, Hug, Martin, Lammertyn, Elise, Landau, Edwina Eddie C, Langley, Ross, Mayer-Hamblett, Nicole, Middleton, Anna, Middleton, Peter, Mielus, Monika, Morrison, Lisa, Munck, Anne, Plant, Barry, Ploeger, Maarten, Pressler, Tacjana, Quon, Bradley S, Radtke, Thomas, Saynor, Zoe L, Shufer, Ilan, Smith, Chris, Smyth, Alan R, Southern, Kevin, van Koningsbruggen-Rietschel, Silke, West, Natalie E   +44 more
core   +7 more sources

Personalized Selection of a CFTR Modulator for a Patient with a Complex Allele [L467F;F508del]

Current Issues in Molecular Biology, 2022
The presence of complex alleles in the CFTR gene can lead to difficulties in diagnosing cystic fibrosis and cause resistance to therapy with CFTR modulators.
Elena Kondratyeva, Nataliya Bulatenko, Yuliya Melyanovskaya, Anna Efremova, Elena Zhekaite, Viktoriya Sherman, Anna Voronkova, Irina Asherova, Alexander Polyakov, Tagui Adyan, Valeriia Kovalskaia, Tatiana Bukharova, Dmitry Goldshtein, Sergey Kutsev   +13 more
doaj   +1 more source

Combined CFTR modulator therapies are linked with anabolic benefits and insulin-sparing in cystic fibrosis-related diabetes

Journal of Clinical & Translational Endocrinology, 2023
Aims: Combined CFTR modulator therapies have dramatically altered pulmonary outcomes in patients with cystic fibrosis (CF). Their impact on glucose metabolism requires further investigations.
Fabian Lurquin, Sophie Gohy, Michel P. Hermans, Vanessa Preumont   +3 more
doaj   +1 more source

Elexacaftor-Tezacaftor-Ivacaftor Reduces Revision Sinus Surgery in People With Cystic Fibrosis. [PDF]

Laryngoscope
Elexacaftor–tezacaftor–ivacaftor (ETI) was associated with a significant decrease in the frequency and rate of endoscopic sinus surgeries in patients with cystic fibrosis. These findings suggest an improvement in CF‐related chronic rhinosinusitis outcomes following ETI and may influence future CF treatment decisions.
Lin A, Komara M, Kistler I, Tan ZH, Lind M, Kotha K, Chiang T.   +6 more
europepmc   +2 more sources

Regulatory T cell enhancement in adults with cystic fibrosis receiving Elexacaftor/Tezacaftor/Ivacaftor therapy

Frontiers in Immunology, 2023
IntroductionCystic fibrosis (CF), especially CF lung disease, is characterized by chronic infection, immune dysfunction including impairment of regulatory T cells (Tregs) and an exaggerated inflammatory response.
Dirk Westhölter, Jonas Raspe, Hendrik Uebner, Johannes Pipping, Mona Schmitz, Svenja Straßburg, Sivagurunathan Sutharsan, Matthias Welsner, Christian Taube, Sebastian Reuter   +9 more
doaj   +1 more source

Extracellular phosphate enhances the function of F508del-CFTR rescued by CFTR correctors [PDF]

, 2021
Background: The clinical response to cystic fibrosis transmembrane conductance regulator (CFTR) modulators varies between people with cystic fibrosis (CF) of the same genotype, in part through the action of solute carriers encoded by modifier genes. Here,
Delpiano, Livia, Gray, Michael A, Lin, JinHeng, Saint-Criq, Vinciane, Sheppard, David N, Wang, Yiting   +5 more
core   +3 more sources

Different CFTR modulator combinations downregulate inflammation differently in cystic fibrosis

eLife, 2020
Previously, we showed that serum and monocytes from patients with CF exhibit an enhanced NLRP3-inflammasome signature with increased IL-18, IL-1β, caspase-1 activity and ASC speck release (Scambler et al. eLife 2019).
Heledd H Jarosz-Griffiths, Thomas Scambler, Chi H Wong, Samuel Lara-Reyna, Jonathan Holbrook, Fabio Martinon, Sinisa Savic, Paul Whitaker, Christine Etherington, Giulia Spoletini, Ian Clifton, Anil Mehta, Michael F McDermott, Daniel Peckham   +13 more
doaj   +1 more source

Effects of CFTR modulators on serum biomarkers of liver fibrosis in children with cystic fibrosis

Hepatology Communications, 2023
The cystic fibrosis (CF) transmembrane conductance regulator corrector/potentiator combinations lumacaftor/ivacaftor and elexacaftor/tezacaftor/ivacaftor improve sweat chloride, pulmonary function, and nutrition.
Steven Levitte, Yonathan Fuchs, Russell Wise, Zachary M. Sellers   +3 more
doaj   +1 more source