Results 21 to 30 of about 6,532 (197)

Treatment patterns in people with cystic fibrosis: have they changed since the introduction of ivacaftor? [PDF]

, 2021
BACKGROUND: In late 2012, ivacaftor became available in the UK for people with cystic fibrosis (CF) aged 6 years and over with a G551D mutation. Long-term changes in treatment patterns have not previously been reported.
Granger, Emily, Keogh, Ruth H, Keogh, RH, Keogh, Ruth H., Davies, G, Granger, E, Davies, Gwyneth   +6 more
core   +1 more source

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

A little CFTR goes a long way: CFTR-dependent sweat secretion from G551D and R117H-5T cystic fibrosis subjects taking ivacaftor. [PDF]

PLoS ONE, 2014
To determine if oral dosing with the CFTR-potentiator ivacaftor (VX-770, Kalydeco) improves CFTR-dependent sweating in CF subjects carrying G551D or R117H-5T mutations, we optically measured sweat secretion from 32-143 individually identified glands in ...
Jessica E Char, Marlene H Wolfe, Hyung-Ju Cho, Il-Ho Park, Jin Hyeok Jeong, Eric Frisbee, Colleen Dunn, Zoe Davies, Carlos Milla, Richard B Moss, Ewart A C Thomas, Jeffrey J Wine   +11 more
doaj   +1 more source

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

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

Long-term impact of Elexacaftor/Tezacaftor/ivacaftor on pulmonary, nutritional and metabolic outcomes in homozygous F508del cystic fibrosis patients: A real-world cohort study. [PDF]

Br J Clin Pharmacol
Aim Elexacaftor/tezacaftor/ivacaftor (ETI) has markedly improved cystic fibrosis (CF) outcomes. However, its long‐term impact on nutrition, metabolism and liver health remains underexplored. We assessed 30‐month changes in pulmonary, nutritional, metabolic and inflammatory markers in people with CF (PwCF) homozygous for F508del.
Perrotta N, Fiorito LA, Vescovo R, Virgilio A, Amato G, Cimino G.   +5 more
europepmc   +2 more sources

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

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

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

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