Results 21 to 30 of about 7,354 (172)

Long-Term Ivacaftor in People Aged 6 Years and Older with Cystic Fibrosis with Ivacaftor-Responsive Mutations

Pulmonary Therapy, 2020
Introduction Mutations in the cystic fibrosis transmembrane conductance regulator gene (CFTR) affect the quantity and/or function of CFTR protein reaching the cell surface.
Joseph M. Pilewski, Kris De Boeck, Jerry A. Nick, Simon Tian, Cynthia DeSouza, Mark Higgins, Richard B. Moss   +6 more
doaj   +1 more source

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

Cystic Fibrosis Care in South Africa: Facing the Challenge of Diversity and Inequality. [PDF]

Pediatr Pulmonol
ABSTRACT Low‐and‐middle‐income countries (LMIC) like South Africa (SA) were left behind with advancements in cystic fibrosis (CF) care that followed re‐imbursement agreements in high‐income countries of CF transmembrane regulator protein modulators (CFTRm) for the treatment of CF.
Zampoli M, Baird C, Morrow B, Verstraete J, Calligaro G, Seale M.   +5 more
europepmc   +2 more sources

Frequency of CFTR variants in southern Brazil and indication for modulators therapy in patients with cystic fibrosis

Genetics and Molecular Biology, 2021
This is a descriptive cross-sectional study that aims to determine the distribution of the CFTR causing variant in a group of patients at a cystic fibrosis (CF) center in southern Brazil, as well as to describe causing variants that are treatable with ...
Eliandra da Silveira Lima, Luíse Sgarabotto Pezzin, Ana Carolina Fensterseifer, Leonardo Araújo Pinto   +3 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

G970R‐CFTR Mutation (c.2908G>C) Results Predominantly in a Splicing Defect

Clinical and Translational Science, 2021
In previous work, participants with a G970R mutation in cystic fibrosis transmembrane conductance regulator (CFTR) (c.2908G>C) had numerically lower sweat chloride responses during ivacaftor treatment than participants with other CFTR gating mutations ...
Meredith C. Fidler, Alexandra Buckley, James C. Sullivan, Marvin Statia, Sylvia F. Boj, Robert G. J. Vries, Anne Munck, Mark Higgins, Matteo Moretto Zita, Paul Negulescu, Fredrick vanGoor, Kris De Boeck   +11 more
doaj   +1 more source

Inhibitory effects of calcium channel blockers nisoldipine and nimodipine on ivacaftor metabolism and their underlying mechanism

Frontiers in Pharmacology
Ivacaftor is the first potentiator of the cystic fibrosis transmembrane conductance regulator (CFTR) protein approved for use alone in the treatment of cystic fibrosis (CF).
Hailun Xia, Xinhao Xu, Jie Chen, Hualu Wu, Yuxin Shen, Xiaohai Chen, Ren-ai Xu, Wenzhi Wu   +7 more
doaj   +1 more source

Impact of CFTR modulator use on outcomes in people with severe cystic fibrosis lung disease

European Respiratory Review, 2020
Drug compounds that augment the production and activity of the cystic fibrosis (CF) transmembrane regulator (CFTR) have revolutionised CF care. Many adults and some children with CF suffer advanced and severe lung disease or await lung transplantation ...
Michal Shteinberg, Jennifer L. Taylor-Cousar   +1 more
doaj   +1 more source

Real-world impact of ivacaftor in people with cystic fibrosis and select ivacaftor-responsive mutations

BMJ Open Respiratory Research
Background Ivacaftor approval was extended to people with cystic fibrosis (CF) with ≥1 of 28 additional ivacaftor-responsive mutations in the USA in 2017 based on preclinical in vitro data.
Mark Higgins, Teja Thorat, Craig McKinnon, Alexander Craft   +3 more
doaj   +1 more source

Ivacaftor in cystic fibrosis adults: Czech experience with six years of follow-up

Biomedical Papers, 2016
Aims: Ivacaftor is a revolutionary treatment option for cystic fibrosis (CF) patients with G551D and other gating mutations. The aim of this study was to evaluate the clinical status of patients on ivacaftor who were followed for up to 6 years together ...
Libor Fila, Lucie Valentova Bartakova, Alzbeta Grandcourtova, Miloslav Marel, Radovan Drnek, Alena Bilkova, Milan Macek, Pavel Drevinek   +7 more
doaj   +1 more source