Results 11 to 20 of about 1,208,918 (256)

Post-approval studies with the CFTR modulators Elexacaftor-Tezacaftor-Ivacaftor. [PDF]

Front Pharmacol, 2023
Triple combination therapy with the CFTR modulators elexacaftor (ELX), tezacaftor (TEZ) and ivacaftor (IVA) has been qualified as a game changer in cystic fibrosis (CF).
Tümmler B.
europepmc   +2 more sources

Neuropsychiatric adverse effects from CFTR modulators deserve a serious research effort. [PDF]

Curr Opin Pulm Med, 2023
Purpose of review This review highlights the problem of neuropsychiatric adverse effects (AEs) associated with elexacaftor/tezacaftor/ivacaftor (ETI), current suboptimal mitigation approaches, a novel testable mechanistic hypothesis, and potential ...
VanElzakker MB, Tillman EM, Yonker LM, Ratai EM, Georgiopoulos AM.   +4 more
europepmc   +2 more sources

The synthetic aminoglycoside ELX-02 induces readthrough of G550X-CFTR producing superfunctional protein that can be further enhanced by CFTR modulators

American Journal of Physiology - Lung cellular and Molecular Physiology, 2023
Ten percent of cystic fibrosis (CF) patients carry a Premature Termination Codon (PTC); no nutation-specific therapies exist for these individuals. ELX-02, a synthetic aminoglycoside, suppresses translation termination at PTCs (i.e., readthrough) by ...
Jianguo Chen, K. Thrasher, Lianwu Fu, Wei Wang, Soheil Aghamohammadzadeh, Hui Wen, Liping Tang, Kim M. Keeling, Emily Falk Libby, David M. Bedwell, Steven M. Rowe   +10 more
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Therapeutic Approaches for Patients with Cystic Fibrosis Not Eligible for Current CFTR Modulators

Cells, 2021
Cystic fibrosis is a severe autosomal recessive disorder caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene encoding the CFTR protein, a chloride channel expressed in many epithelial cells.
Isabelle Fajac, Isabelle Sermet
doaj   +2 more sources

Impact of CFTR Modulators on the Impaired Function of Phagocytes in Cystic Fibrosis Lung Disease [PDF]

International Journal of Molecular Sciences, 2022
Cystic fibrosis (CF), the most common genetically inherited disease in Caucasian populations, is a multi-systemic life-threatening autosomal recessive disorder caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. In
Aniello Meoli, Olaf Eickmeier, Giovanna Pisi, Valentina Fainardi, Stefan Zielen, Susanna Esposito   +5 more
openalex   +2 more sources

Experience to date with CFTR modulators during pregnancy and breastfeeding in the British Columbia Cystic Fibrosis clinic

Respiratory Medicine Case Reports, 2022
The introduction and rapid uptake of CFTR modulator therapy, in addition to other treatments, has significantly increased life expectancy in CF and provided more women the opportunity to consider and successfully be managed throughout pregnancy. There is
Jodi Goodwin, Bradley S. Quon, Pearce G. Wilcox   +2 more
doaj   +2 more sources

Revisiting Host-Pathogen Interactions in Cystic Fibrosis Lungs in the Era of CFTR Modulators. [PDF]

Int J Mol Sci, 2023
Cystic fibrosis transmembrane conductance regulator (CFTR) modulators, a new series of therapeutics that correct and potentiate some classes of mutations of the CFTR, have provided a great therapeutic advantage to people with cystic fibrosis (pwCF).
Ribeiro CMP, Higgs MG, Muhlebach MS, Wolfgang MC, Borgatti M, Lampronti I, Cabrini G.   +6 more
europepmc   +2 more sources

Inflammatory Activity of Epithelial Stem Cell Variants from Cystic Fibrosis Lungs Is Not Resolved by CFTR Modulators. [PDF]

Am J Respir Crit Care Med, 2023
Rationale CFTR (cystic fibrosis transmembrane conductance regulator) modulator drugs restore function to mutant channels in patients with cystic fibrosis (CF) and lead to improvements in body mass index and lung function.
Wang S, Niroula S, Hoffman A, Khorrami M, Khorrami M, Yuan F, Gasser GN, Choi S, Liu B, Li J, Metersky ML, Vincent M, Crum CP, Boucher RC, Karmouty-Quintana H, Huang HJ, Sheshadri A, Dickey BF, Parekh KR, Engelhardt JF, McKeon FD, Xian W.   +21 more
europepmc   +2 more sources

Lived experiences of individuals with cystic fibrosis on CFTR-modulators

BMC Pulmonary Medicine, 2022
Background CFTR-modulators are a category of drugs that facilitate trafficking and opening of the abnormal CFTR protein in individuals with cystic fibrosis (CF) who have certain genetic mutations.
Annelise Page, Aaron Goldenberg, Anne L. Matthews   +2 more
doaj   +2 more sources

Nutritional impact of CFTR modulators in children with cystic fibrosis. [PDF]

Front Pediatr, 2023
Background Nutritional status is a major prognostic factor for breathing and the survival of patients with cystic fibrosis (CF). Since 2012, the development of CFTR modulators has considerably transformed the outcome of this disease.
Gaschignard M, Beaufils F, Lussac-Sorton F, Gallet P, Clouzeau H, Menard J, Costanzo A, Nouard L, Delhaes L, Tetard C, Lamireau T, Fayon M, Bui S, Enaud R.   +13 more
europepmc   +2 more sources