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Left Behind: The Potential Impact of CFTR Modulators on Racial and Ethnic Disparities in Cystic Fibrosis [PDF]
Paediatric Respiratory Reviews, 2021 The advent of CFTR modulators, a genomic specific medication, revolutionized the treatment of CF for many patients. However, given that these therapeutics were only developed for specific CFTR mutations, not all people with CF have access to such disease-
M. McGarry +3 more
semanticscholar +2 more sources
Frontiers in Molecular Biosciences, 2023 Most of the 2,100 CFTR gene variants reported to date are still unknown in terms of their disease liability in Cystic Fibrosis (CF) and their molecular and cellular mechanism that leads to CFTR dysfunction.
Violeta Railean +7 more
doaj +2 more sources
Orphanet Journal of Rare Diseases, 2022 Background Over the past decade, a new class of drugs called CFTR (cystic fibrosis transmembrane conductance regulator) modulators have shown to be able to improve clinical outcomes in patient with Cystic Fibrosis.
Enrico Costa +4 more
doaj +2 more sources
CFTR Modulators: The Changing Face of Cystic Fibrosis in the Era of Precision Medicine
Frontiers in Pharmacology, 2020 Cystic fibrosis (CF) is a lethal inherited disease caused by mutations in the CF transmembrane conductance regulator (CFTR) gene, which result in impairment of CFTR mRNA and protein expression, function, stability or a combination of these.
Miquéias Lopes-Pacheco
doaj +2 more sources
Strategies to investigate the mechanism of action of CFTR modulators [PDF]
Journal of Cystic Fibrosis, 2004 The malfunction of the cystic fibrosis transmembrane conductance regulator (CFTR) Cl- channel is associated with a wide spectrum of disease. In the search for modulators of CFTR, pharmaceutical agents have been identified that (i) act indirectly by regulating the protein kinases and phosphatases, which control CFTR, and (ii) interact directly with CFTR.
Cai, Zhiwei +4 more
openaire +3 more sources
The Effect of CFTR Modulators on Airway Infection in Cystic Fibrosis
International Journal of Molecular Sciences, 2022 The advent of Cystic fibrosis transmembrane receptor (CFTR) modulators in 2012 was a critical event in the history of cystic fibrosis (CF) treatment. Unlike traditional therapies that target downstream effects of CFTR dysfunction, CFTR modulators aim to ...
Caitlyn Harvey +4 more
semanticscholar +2 more sources
Scientific Reports, 2017 Clinical studies with modulators of the Cystic Fibrosis Transmembrane conductance Regulator (CFTR) protein have demonstrated that functional restoration of the mutated CFTR can lead to substantial clinical benefit.
Iwona M. Pranke +17 more
doaj +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 +2 more
doaj +2 more sources
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 +6 more sources
Robust Stimulation of W1282X-CFTR Channel Activity by a Combination of Allosteric Modulators. [PDF]
PLoS ONE, 2016 W1282X is a common nonsense mutation among cystic fibrosis patients that results in the production of a truncated Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) channel. Here we show that the channel activity of the W1282X-CFTR polypeptide is
Wei Wang +4 more
doaj +2 more sources