Results 21 to 30 of about 7,376 (199)

A descriptive case series of hepatotoxicity associated with CFTR modulators and possible relevance of pharmacogenetic polymorphisms in cystic fibrosis patients. [PDF]

Br J Clin Pharmacol
Cystic fibrosis transmembrane conductance regulator (CFTR) modulators are widely used in patients with cystic fibrosis and significantly improve respiratory function and quality of life. However, their effectiveness may be limited by liver damage, which sometimes leads to treatment discontinuation, and the mechanisms underlying this remain poorly ...
Laffitte Redondo C, Bourdin V, Ait Tayeb AEK, Benaboud S, Martins I, Joyau C, Benhamida M, Sermet-Gaudelus I, Martin C, Burgel PR, Verstuyft C, Chouchana L, Ayme-Dietrich E.   +12 more
europepmc   +2 more sources

Weight increase in people with cystic fibrosis on CFTR modulator therapy is mainly due to increase in fat mass

Frontiers in Pharmacology, 2023
Background: Ivacaftor, the first CFTR modulator drug, leads to significant long-term improvement in lung function and weight gain. The mechanism as well as the long-term impact of ivacaftor on weight, resting energy expenditure (REE) and body composition
Marialena Mouzaki, Annie Dupuis, Julie Avolio, Katherine Griffin, Felix Ratjen, Elizabeth Tullis, Tanja Gonska   +6 more
doaj   +1 more source

Antibacterial properties of the CFTR potentiator ivacaftor [PDF]

, 2014
BackgroundIvacaftor increases CFTR channel activity and improves pulmonary function for individuals bearing a G551D mutation. Because ivacaftor structurally resembles quinolone antibiotics, we tested the hypothesis that ivacaftor possesses antibacterial ...
Gansemer, Nick D., Abou Alaiwa, Mahmoud H., Dohrn, Cassie L., Welsh, Michael J., Diekema, Daniel J., Reznikov, Leah R., Stoltz, David A.   +6 more
core   +1 more source

Tezacaftor-ivacaftor in residual-function heterozygotes with cystic fibrosis [PDF]

, 2017
BACKGROUND Cystic fibrosis is an autosomal recessive disease caused by mutations in the CFTR gene that lead to progressive respiratory decline. Some mutant CFTR proteins show residual function and respond to the CFTR potentiator ivacaftor in vitro ...
Han, Linda, Ingenito, Edward P., Wilson, John, Ringshausen, Felix C., Kerem, Eitan, Ringshausen, Felix C, Ingenito, Edward P, Cori Daines, Felix C. Ringshausen, Edward P. Ingenito, Steven M. Rowe, John Wilson, Rowe, Steven M, Simard, Christopher, Elizabeth Tullis, Julie Lekstrom-Himes, Christopher Simard, Jane C. Davies, Charlotte McKee, Davies, Jane C, Mckee, Charlotte, Tullis, Elizabeth, Linda Han, Nair, Nitin, Eitan Kerem, Lekstrom-Himes, Julie, Davies, Jane C., Nitin Nair, Daines, Cori, Rowe, Steven M.   +29 more
core   +1 more source

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

Ivacaftor potentiation of multiple CFTR channels with gating mutations [PDF]

, 2012
BackgroundThe investigational CFTR potentiator ivacaftor (VX-770) increased CFTR channel activity and improved lung function in subjects with CF who have the G551D CFTR gating mutation.
Johnson, James P., Sussky, Katherine, Hoffman, Beth J., Joubran, John, Yu, Haihui, Huang, Chien-Jung, Urrutia, Art, Seepersaud, Sheila, Van Goor, Fredrick, Worley, Jennings, Cao, Dong, Burton, Bill   +11 more
core   +1 more source

Elexacaftor-Tezacaftor-Ivacaftor and Ivacaftor (Trikafta)

Canadian Journal of Health Technologies, 2021
CADTH reimbursement reviews are comprehensive assessments of the clinical effectiveness and cost-effectiveness, as well as patient and clinician perspectives, of a drug or drug class. The assessments inform non-binding recommendations that help guide the reimbursement decisions of Canada's federal, provincial, and territorial governments ...
openaire   +3 more sources

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

Ivacaftor Inhibits Glioblastoma Stem Cell Maintenance and Tumor Progression

Frontiers in Cell and Developmental Biology, 2021
Glioblastoma (GBM) is the most common and malignant primary brain tumor. Glioblastoma stem cells (GSCs) not only initiate and sustain uncontrolled cell proliferation but also resistant to conventional clinical therapies including temozolomide (TMZ ...
Kun Liu, Kun Liu, Jun Pu, Zhi Nie, Zhi Nie, Yulin Shi, Yulin Shi, Liping Jiang, Qisheng Wu, Yongbin Chen, Yongbin Chen, Yongbin Chen, Cuiping Yang, Cuiping Yang   +13 more
doaj   +1 more source

The rescue of F508del-CFTR by elexacaftor/tezacaftor/ivacaftor (Trikafta) in human airway epithelial cells is underestimated due to the presence of ivacaftor

, 2021
International audienceTrikafta, currently the leading therapeutic in Cystic Fibrosis (CF), has demonstrated a real clinical benefit. This treatment is the triple combination therapy of two folding correctors elexacaftor/tezacaftor (VX445/VX661) plus the ...
Mirval, Sandra, Carrez, Thomas, Coraux, Christelle, Lévêque, Manuella, Cantereau, Anne, Sage, Edouard, Billet, Arnaud, Becq, Frédéric   +7 more
core   +1 more source