Heterogeneity of weight gain after initiation of Elexacaftor/Tezacaftor/Ivacaftor in people with cystic fibrosis [PDF]
, 2023 Background: The introduction of the novel therapy, Elexacaftor/Tezacaftor/Ivacaftor (ETI) has been effective in improving weight gain in both clinical trials and real-world studies. However, the magnitude of this effect appears to be heterogeneous across
Aliberti, Stefano +9 more
core +1 more source
Personalized Selection of a CFTR Modulator for a Patient with a Complex Allele [L467F;F508del]
Current Issues in Molecular Biology, 2022 The presence of complex alleles in the CFTR gene can lead to difficulties in diagnosing cystic fibrosis and cause resistance to therapy with CFTR modulators.
Elena Kondratyeva +13 more
doaj +1 more source
Journal of Clinical & Translational Endocrinology, 2023 Aims: Combined CFTR modulator therapies have dramatically altered pulmonary outcomes in patients with cystic fibrosis (CF). Their impact on glucose metabolism requires further investigations.
Fabian Lurquin +3 more
doaj +1 more source
Respiratory Oscillometry and Multimodal Lung Function Assessment of Elexacaftor/Tezacaftor/Ivacaftor Response in Children and Young Adults With Cystic Fibrosis. [PDF]
Pediatr PulmonolABSTRACT Background To date, there is no data on forced oscillation technique (FOT) as a tool to evaluate elexacaftor/tezacaftor/ivacaftor (ETI) modulator in young patients with cystic fibrosis (pwCF). Objective Our study aimed to assess the effect of a 6‐month ETI treatment on FOT parameters and compare it to the effect on spirometry and nitrogen ...
Kogias C +8 more
europepmc +2 more sources
Different CFTR modulator combinations downregulate inflammation differently in cystic fibrosis
eLife, 2020 Previously, we showed that serum and monocytes from patients with CF exhibit an enhanced NLRP3-inflammasome signature with increased IL-18, IL-1β, caspase-1 activity and ASC speck release (Scambler et al. eLife 2019).
Heledd H Jarosz-Griffiths +13 more
doaj +1 more source
Worldwide rates of diagnosis and effective treatment for cystic fibrosis [PDF]
, 2022 BACKGROUND: Time has seen management for Cystic Fibrosis (CF) advance drastically, most recently in the development of the disease-modifying triple combination therapy ivacaftor/tezacaftor/elexacaftor.
Garratt, A, Guo, J, Hill, A
core +1 more source
Effects of CFTR modulators on serum biomarkers of liver fibrosis in children with cystic fibrosis
Hepatology Communications, 2023 The cystic fibrosis (CF) transmembrane conductance regulator corrector/potentiator combinations lumacaftor/ivacaftor and elexacaftor/tezacaftor/ivacaftor improve sweat chloride, pulmonary function, and nutrition.
Steven Levitte +3 more
doaj +1 more source
Standards of care for CFTR variant-specific therapy (including modulators) for people with cystic fibrosis [PDF]
, 2023 Cystic fibrosis; Guidelines; Variant-specific therapyFibrosis quística; Pautas; Terapia variante específicaFibrosi quística; Pautes; Teràpia variant específicaCystic fibrosis (CF) has entered the era of variant-specific therapy, tailored to the genetic ...
Castellani, Carlo +6 more
core +1 more source
Pharmacokinetic and Drug–Drug Interaction Profiles of the Combination of Tezacaftor/Ivacaftor
Clinical and Translational Science, 2019 Drug–drug interaction (DDI) studies are described for tezacaftor/ivacaftor, a new cystic fibrosis transmembrane conductance regulator modulator therapy for the treatment of cystic fibrosis.
Varun Garg +11 more
doaj +1 more source
New era of cystic fibrosis: full mutational analysis and personalized therapy [PDF]
, 2017 Despite its apparently simple genetics, cystic fibrosis (CF) is a rather complex genetic disease. A lot of variability in the steps of the path from the cystic fibrosis transmembrane conductance regulator (CFTR ) gene to the clinical manifestations ...
Lucarelli, Marco
core +1 more source