Results 41 to 50 of about 9,288 (216)
Children, 2023 Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) is a new CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) modulator treatment, used over the last few years, which has shown an improvement in different clinical outcomes in patients with cystic ...
Nikoletta Kapouni +3 more
doaj +1 more source
Elexacaftor–Tezacaftor–Ivacaftor Improves Sinonasal Outcomes in Young Children With Cystic Fibrosis
International Forum of Allergy and RhinologySevere chronic rhinosinusitis (CRS) is a near universal manifestation of cystic fibrosis. Elexacaftor/tezacaftor/ivacaftor (ETI) is an oral, small molecule, highly effective Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) corrector–potentiator
Amanda L. Stapleton +10 more
semanticscholar +1 more source
British Journal of Clinical Pharmacology, EarlyView.Cystic fibrosis transmembrane conductance regulator (CFTR) modulators are widely used in patients with cystic fibrosis and significantly improve respiratory function and quality of life. However, their effectiveness may be limited by liver damage, which sometimes leads to treatment discontinuation, and the mechanisms underlying this remain poorly ...
Clara Laffitte Redondo +12 more
wiley +1 more source
SLC26A9 Gene Is Associated With Lung Function Response to Ivacaftor in Patients With Cystic Fibrosis
Frontiers in Pharmacology, 2018 Ivacaftor is a drug used to treat cystic fibrosis (CF) patients carrying specific gating CFTR mutations. Interpatient variability in the lung response has been shown to be partly explained by rs7512462 in the Solute Carrier Family 26 Member 9 (SLC26A9 ...
Harriet Corvol +7 more
doaj +1 more source
G970R‐CFTR Mutation (c.2908G>C) Results Predominantly in a Splicing Defect
Clinical and Translational Science, 2021 In previous work, participants with a G970R mutation in cystic fibrosis transmembrane conductance regulator (CFTR) (c.2908G>C) had numerically lower sweat chloride responses during ivacaftor treatment than participants with other CFTR gating mutations ...
Meredith C. Fidler +11 more
doaj +1 more source
Frontiers in ImmunologyBackground CFTR modulator therapies have positive clinical outcomes, yet chronic inflammation and bacterial infections persist in people with CF (pwCF).
H. Ozuna +12 more
semanticscholar +1 more source
Regulatory harmonization: Evolution, globalization and future directions
British Journal of Clinical Pharmacology, EarlyView.Regulatory harmonization has become an increasingly important and accepted approach to streamline regulatory review processes and expedite access to safe, effective and high‐quality medicines globally. This review explores the evolution and current status of regulatory harmonization, convergence and reliance initiatives.
Orin Chisholm +2 more
wiley +1 more source
Ivacaftor in cystic fibrosis adults: Czech experience with six years of follow-up
Biomedical Papers, 2016 Aims: Ivacaftor is a revolutionary treatment option for cystic fibrosis (CF) patients with G551D and other gating mutations. The aim of this study was to evaluate the clinical status of patients on ivacaftor who were followed for up to 6 years together ...
Libor Fila +7 more
doaj +1 more source
The Lancet, 2019 Summary Background Cystic fibrosis transmembrane conductance regulator (CFTR) modulators correct the basic defect caused by CFTR mutations. Improvements in health outcomes have been achieved using the combination of a CFTR corrector and potentiator in ...
H. Heijerman +19 more
semanticscholar +1 more source
JPGN Reports, EarlyView.Abstract Objectives The efficacy of cystic fibrosis transmembrane conductance regulator (CFTR)‐modulator therapies in preventing or ameliorating cystic fibrosis liver disease (CFLD) by correcting CFTR in cholangiocytes is not well‐documented. This study aimed to assess liver function during CFTR‐modulators.
Laura Giugliano +12 more
wiley +1 more source