Results 51 to 60 of about 4,690 (200)

Divergent neurobehavioral effects of CFTR modulators elexacaftor and ivacaftor in mice

Acta Psychologica
Recent advances in cystic fibrosis transmembrane conductance regulator (CFTR) modulator combination therapies have markedly improved survival and quality of life for people with cystic fibrosis (CF).
Qian Ge, Amy Fagan, Hui Lu, Jianyang Du, Leah R. Reznikov   +4 more
doaj   +1 more source

Elexacaftor-Tezacaftor-Ivacaftor in 2 cystic fibrosis adults homozygous for M1101K with end-stage lung disease

Respiratory Medicine Case Reports, 2023
Elexacaftor-tezacaftor-ivacaftor (ETI) therapy is shown to improve the health of individuals with cystic fibrosis (CF) who have the F508del variant. There are in vitro studies showing benefit with ETI for select rare CF variants.
Winnie M. Leung, Parastoo Molla Davoodi, Ashten Langevin, Clare Smith, Michael D. Parkins   +4 more
doaj   +1 more source

Impact of Elexacaftor–Tezacaftor–Ivacaftor on lung disease in cystic fibrosis

Pediatric Pulmonology, 2023
AbstractBackgroundIn people with cystic fibrosis (pwCF), the impact of cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies, such as Elexacaftor–Tezacaftor–Ivacaftor (ETI), on structural changes in the lungs is unclear.ObjectiveTo determine the impact of ETI on clinical parameters and on structural lung disease as measured by ...
Courtney Gushue, Mariah Eisner, Shasha Bai, Terri Johnson, Melissa Holtzlander, Karen McCoy, Shahid Sheikh   +6 more
openaire   +2 more sources

Organic Synthesis and Current Understanding of the Mechanisms of CFTR Modulator Drugs Ivacaftor, Tezacaftor, and Elexacaftor

Molecules
The monogenic rare disease Cystic Fibrosis (CF) is caused by mutations in the gene encoding the CF transmembrane conductance (CFTR) protein, an anion channel expressed at the apical plasma membrane of epithelial cells.
Filipa C. Ferreira, Camilla D. Buarque, Miquéias Lopes-Pacheco   +2 more
doaj   +1 more source

Precision Medicine Based on CFTR Genotype for People with Cystic Fibrosis

Pharmacogenomics and Personalized Medicine, 2022
Iram Haq,1,2 Maryam Almulhem,1 Simone Soars,1 David Poulton,2,3 Malcolm Brodlie1,2 1Translational and Clinical Research Institute, Faculty of Medical Sciences, Newcastle University, Newcastle upon Tyne, UK; 2Paediatric Respiratory Medicine, Great North ...
Haq I, Almulhem M, Soars S, Poulton D, Brodlie M   +4 more
doaj  

Clinical efficacy of elexacaftor-tezacaftor-ivacaftor in an adolescent with homozygous G85E cystic fibrosis

Respiratory Medicine Case Reports, 2022
Elexacaftor-tezacaftor-ivacaftor (ELX/TEZ/IVA) is a triple combination drug therapy approved for individuals with cystic fibrosis (CF) who possess at least one copy of the F508del cystic fibrosis transmembrane conductance regulator (CFTR) variant.
Elena Stekolchik, David Saul, Aaron Chidekel   +2 more
doaj   +1 more source

Impact of elexacaftor–tezacaftor–ivacaftor on bacterial colonization and inflammatory responses in cystic fibrosis

Pediatric Pulmonology, 2022
Cystic fibrosis (CF) is a multisystem disease with progressive deterioration. Recently, CF transmembrane conductance regulator (CFTR) modulator therapies were introduced that repair underlying protein defects. Objective of this study was to determine the
S. Sheikh, R. Britt, N. Ryan-Wenger, Aiman Q. Khan, B. Lewis, Courtney Gushue, Hazel Ozuna, Devi Jaganathan, K. McCoy, B. Kopp   +9 more
semanticscholar   +1 more source

Elexacaftor–Tezacaftor–Ivacaftor Therapy for Cystic Fibrosis Patients with The F508del/Unknown Genotype

Antibiotics, 2021
The new CFTR modulator combination, elexacaftor/tezacaftor/ivacaftor (Trikafta) was approved by the FDA in October 2019 for treatment of Cystic Fibrosis in patients 6 years of age or older who have at least one F508del mutation in one allele and a ...
Marika Comegna, Vito Terlizzi, Donatello Salvatore, Carmela Colangelo, Antonella Miriam Di Lullo, Immacolata Zollo, Giovanni Taccetti, Giuseppe Castaldo, Felice Amato   +8 more
doaj   +1 more source

Rethinking hyperbilirubinemia: Gilbert syndrome in children with cystic fibrosis, a case report

Respiratory Medicine Case Reports
Cystic Fibrosis leads to liver complications, including cystic fibrosis liver disease but hyperbilirubinemia in CF patients on CFTR modulators is less understood.
Yara Salameh, John Lyles, Shatha Yousef
doaj   +1 more source

Elexacaftor/tezacaftor/ivacaftor and inflammation in children and adolescents with cystic fibrosis: a retrospective dual-center cohort study

Therapeutic Advances in Respiratory Disease
Background: Cystic fibrosis (CF) is characterized by chronic neutrophilic inflammation in the airways. Elexacaftor/tezacaftor/ivacaftor (ETI) therapy has demonstrably improved clinical outcomes and quality of life in people with CF (pwCF), but its ...
Angela Pepe, C. Fevola, D. Dolce, S. Campana, N. Ravenni, G. Taccetti, D. Salvatore, V. Terlizzi   +7 more
semanticscholar   +1 more source