Results 71 to 80 of about 6,985 (222)

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

, 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.
Balmpouzis, Zisis, Baud, David, Faure van Rossum, Annabelle, Koutsokera, Angela, Mazza Stalder, Jesica, Mitropoulou, Georgia, Panchaud, Alice   +6 more
core   +3 more sources

Improved Quality of Life in Children With Cystic Fibrosis Who Received Transmembrane Conductance Regulator Modulators

Acta Paediatrica, EarlyView.
ABSTRACT Aim Children with cystic fibrosis (CF) face substantial daily treatment burdens and the effects of transmembrane conductance regulator modulators on these have not been sufficiently described. We evaluated changes in treatment burden after elexacaftor tezacaftor ivacaftor (ETI) was initiated.
Marcus Svedberg, Frida Lundqvist, Huda Abdulahi Östrand, Christina Krantz, Per Ekman, Henrik Imberg   +5 more
wiley   +1 more source

Longitudinal profiling of the intestinal microbiome in children with cystic fibrosis treated with elexacaftor-tezacaftor-ivacaftor

mBio
The intestinal microbiome influences growth and disease progression in children with cystic fibrosis (CF). Elexacaftor-tezacaftor-ivacaftor (ELX/TEZ/IVA), the newest pharmaceutical modulator for CF, restores the function of the pathogenic mutated CF ...
Seth A. Reasoner, Rachel Bernard, Adam Waalkes, Kelsi Penewit, Janessa Lewis, Andrew G. Sokolow, Rebekah F. Brown, Kathryn M. Edwards, Stephen J. Salipante, Maria Hadjifrangiskou, Maribeth R. Nicholson   +10 more
doaj   +1 more source

Qualitative and quantitative evaluation of computed tomography changes in adults with cystic fibrosis treated with elexacaftor-tezacaftor-ivacaftor: a retrospective observational study [PDF]

, 2023
Introduction: The availability of highly effective triple cystic fibrosis transmembrane conductance regulator (CFTR) modulator combination therapy with elexacaftor-tezacaftor-ivacaftor (ETI) has improved pulmonary outcomes and quality of life of people ...
Dettmer, Sabine, Fuge, Jan, Lammers, Oliver, Ringshausen, Felix C., Sauer-Heilborn, Annette, Wacker, Frank, Weinheimer, Oliver, Welte, Tobias, Wielpütz, Mark O   +8 more
core   +1 more source

Clinical development of triple-combination CFTR modulators for cystic fibrosis patients with one or two F508del alleles

ERJ Open Research, 2019
Cystic fibrosis (CF) is caused by mutations in the CF transmembrane conductance regulator gene (CFTR) that result in diminished quantity and/or function of the CFTR anion channel.
Jennifer L. Taylor-Cousar, Marcus A. Mall, Bonnie W. Ramsey, Edward F. McKone, Elizabeth Tullis, Gautham Marigowda, Charlotte M. McKee, David Waltz, Samuel M. Moskowitz, Jessica Savage, Fengjuan Xuan, Steven M. Rowe   +11 more
doaj   +1 more source

Molecular basis of cystic fibrosis and modern therapeutic approaches [PDF]

, 2019
Identifikacija molekularne povezanosti između genske nefunkcionalnosti CFTR gena te patofiziološke i kliničke pozadine cistične fibroze omogućila je razvoj suvremenih terapijskih modela koji su primarno utemeljeni na individualiziranom pristupu ...
Dragičević, Dorian
core   +2 more sources

EPS5.04 Liver test abnormalities in adults with cystic fibrosis taking elexacaftor/tezacaftor/ivacaftor – one size doesn’t fit all? [PDF]

, 2023
D. Tewkesbury, Varinder Athwal, R. Bright-Thomas, Andrew Jones, Peter Barry   +4 more
openalex   +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

Pharmacokinetics of Ivacaftor, Tezacaftor, Elexacaftor, and Lumacaftor in Special Cystic Fibrosis Populations:A Systematic Review [PDF]

BACKGROUND AND OBJECTIVE: Following the development of cystic fibrosis transmembrane conductance regulator (CFTR) modulators (ivacaftor, tezacaftor, elexacaftor, and lumacaftor), the prognosis for people diagnosed with cystic fibrosis (pwCF) has improved.
Akkerman, Onno W, Elzinga, Femke A, Koppelman, Gerard H, Malik, Paul R V, Mian, Paola, Rottier, Bart L, Touw, Daan J, van der Vaart, Hester   +7 more
core   +1 more source

P329 Audit of change in weight and BMI centile in children on elexacaftor/tezacaftor/ivacaftor in the Liverpool paediatric cystic fibrosis population [PDF]

, 2023
Clare Woodland, C.A. Berry
openalex   +1 more source