Results 41 to 50 of about 7,856 (243)

Cystic Fibrosis Patients of Minority Race and Ethnicity Less Likely Eligible for CFTR Modulators Based on CFTR Genotype

Pediatric Pulmonology, 2021
Cystic fibrosis transmembrane conductance regulator (CFTR) modulators are disease‐modifying medications for cystic fibrosis (CF) and are shown to be efficacious for only specific CFTR mutations.
M. McGarry, S. McColley
semanticscholar   +1 more source

Enhanced Expression of Human Epididymis Protein 4 (HE4) Reflecting Pro-Inflammatory Status Is Regulated by CFTR in Cystic Fibrosis Bronchial Epithelial Cells

Frontiers in Pharmacology, 2021
Decreased human epididymis protein 4 (HE4) plasma levels were reported in cystic fibrosis (CF) patients under CFTR potentiator ivacaftor therapy, which inversely correlated with lung function improvement.
Zsolt Bene, Zsolt Bene, Zsolt Bene, Zsolt Fejes, Zsolt Fejes, Tibor Gabor Szanto, Ferenc Fenyvesi, Judit Váradi, Luka A. Clarke, Gyorgy Panyi, Milan Macek, Margarida D. Amaral, István Balogh, István Balogh, Béla Nagy, Béla Nagy   +15 more
doaj   +1 more source

Neuropsychiatric adverse effects from CFTR modulators deserve a serious research effort

Current opinion in pulmonary medicine, 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 ...
M. VanElzakker, E. Tillman, L. Yonker, E. Ratai, A. Georgiopoulos   +4 more
semanticscholar   +1 more source

Cystic fibrosis liver disease in the new era of cystic fibrosis transmembrane conductance receptor (CFTR) modulators.

Paediatric Respiratory Reviews, 2023
Cystic fibrosis liver disease (CFLD) is characterised by a wide heterogenity of manifestations and severity. It represents a major cause of morbidity in people with cystic fibrosis (PwCF), which will be of increasing relevance as survival increases in ...
Jessica A Eldredge, M. Oliver, Chee Y. Ooi   +2 more
semanticscholar   +1 more source

Modulation of CFTR gating by permeant ions [PDF]

Journal of General Physiology, 2014
Cystic fibrosis transmembrane conductance regulator (CFTR) is unique among ion channels in that after its phosphorylation by protein kinase A (PKA), its ATP-dependent gating violates microscopic reversibility caused by the intimate involvement of ATP hydrolysis in controlling channel closure. Recent studies suggest a gating model featuring an energetic
Yeh, Han-I, Yeh, Jiunn-Tyng, Hwang, Tzyh-Chang   +2 more
openaire   +2 more sources

Nutritional impact of CFTR modulators in children with cystic fibrosis

Frontiers in Pediatrics, 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.
M. Gaschignard, F. Beaufils, Florian Lussac-Sorton, P. Gallet, H. Clouzeau, Joris Menard, Aurélie Costanzo, Lucie Nouard, L. Delhaes, C. Tétard, T. Lamireau, M. Fayon, S. Bui, R. Enaud   +13 more
semanticscholar   +1 more source

S945L-CFTR molecular dynamics, functional characterization and tezacaftor/ivacaftor efficacy in vivo and in vitro in matched pediatric patient-derived cell models

Frontiers in Pediatrics, 2022
Cystic Fibrosis (CF) results from over 400 different disease-causing mutations in the CF Transmembrane Conductance Regulator (CFTR) gene. These CFTR mutations lead to numerous defects in CFTR protein function.
Katelin M. Allan, Katelin M. Allan, Miro A. Astore, Laura K. Fawcett, Laura K. Fawcett, Laura K. Fawcett, Sharon L. Wong, Sharon L. Wong, Po-Chia Chen, Renate Griffith, Adam Jaffe, Adam Jaffe, Adam Jaffe, Serdar Kuyucak, Shafagh A. Waters, Shafagh A. Waters, Shafagh A. Waters, Shafagh A. Waters   +17 more
doaj   +1 more source

Cystic Fibrosis: Treatment with CFTR Modulators

, 2023
CFTR modulators have been a groundbreaking new invention starting with the approval of the CFTR corrector Ivacaftor in 2012. Nowadays, up to 90% of CF patients have the potential ability to get treated by an appropriate CFTR modulator. Ivacaftor showed
Westphal, Anna Philine
core   +2 more sources

Current prices versus minimum costs of production for CFTR modulators

medRxiv, 2022
Background: While the clinical benefits of CFTR modulators are clear, their high prices render them inaccessible outside of a select few countries. Despite this, there is currently limited evidence regarding access to these transformative therapies ...
Jonathan Guo, Junzheng Wang, Jingchun Zhang, J. Fortunak, A. Hill   +4 more
semanticscholar   +1 more source

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

American Journal of Respiratory and Critical Care Medicine, 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.
S. Wang, S. Niroula, Ashley Hoffman, Melika Khorrami, M. Khorrami, Feng Yuan, Grace N. Gasser, Soon H. Choi, Bovey Liu, Justin Li, M. Metersky, Matthew J. Vincent, Christopher P. Crum, R. C. Boucher, H. Karmouty-Quintana, Howard Huang, A. Sheshadri, B. Dickey, K. Parekh, J. Engelhardt, Frank D McKeon, Wa Xian   +21 more
semanticscholar   +1 more source