Hyperbilirubinemia and Gilbert's syndrome in Cystic Fibrosis patients treated with elexacaftor/tezacaftor/ivacaftor [PDF]
, 2023 Vito Terlizzi +10 more
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Rethinking hyperbilirubinemia: Gilbert syndrome in children with cystic fibrosis, a case report
Respiratory Medicine Case ReportsCystic Fibrosis leads to liver complications, including cystic fibrosis liver disease but hyperbilirubinemia in CF patients on CFTR modulators is less understood.
Yara Salameh, John Lyles, Shatha Yousef
doaj +1 more source
Evaluation of the response to elexacaftor-tezacaftor-ivacaftor of the rare CFTR variants L383S, I507del, L1065P and R1066H in intestinal organoid-derived epithelial monolayers [PDF]
Introduction: Cystic fibrosis (CF) is caused by mutation of the CFTR gene, encoding an epithelial anion channel. Here we evaluated the effect of the modulator combination elexacaftor-tezacaftor-ivacaftor (ETI) on the function of four rare, poorly ...
Angyal, Dora +16 more
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A case of severe pulmonary exacerbation in a young patient with cystic fibrosis in the era of CFTR modulators [PDF]
The introduction of CFTR modulator drugs like Elexacaftor-Tezacaftor-Ivacaftor (ETI) has transformed the management of Cystic Fibrosis (CF), significantly improving symptoms, lung function, and quality of life, while reducing reliance on intravenous ...
Alicandro, Gianfranco +8 more
core +1 more source
Therapeutic drug monitoring-guided dosing for pediatric cystic fibrosis patients: recent advances and future outlooks [PDF]
, 2023 Introduction: Medicine use in children with cystic fibrosis (CF) is complicated by inconsistent pharmacokinetics at variance with the general population, a lack of research into this and its effects on clinical outcomes.
Bentley, Sian +5 more
core
BMC Pulmonary MedicineBackground Physical activity is a crucial demand on cystic fibrosis treatment management. The highest value of oxygen uptake (VO2peak) is an appropriate tool to evaluate the physical activity in these patients.
Nela Stastna +6 more
doaj +1 more source
WS06.04 Lung volumes as possible factors explaining the heterogeneity in the response to elexacaftor/tezacaftor/ivacaftor in patients with cystic fibrosis and advanced lung disease [PDF]
, 2023 Chiara Premuda +8 more
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Vanzacaftor–tezacaftor–deutivacaftor for children aged 6–11 years with cystic fibrosis (RIDGELINE Trial VX21-121-105): an analysis from a single-arm, phase 3 trial [PDF]
BACKGROUND: In phase 2 trials in people with cystic fibrosis aged 18 years and older, vanzacaftor–tezacaftor–deutivacaftor has been shown to be a safe and effective, once-daily cystic fibrosis transmembrane conductance regulator (CFTR) modulator ...
Chen, YC +17 more
core
Pharmacological rescue of the G85E CFTR variant by preclinical and approved modulators
Frontiers in PharmacologyIntroductionCystic Fibrosis (CF) is a genetic disease due to loss-of-function mutations of the CFTR channel. F508del is the most frequent mutation (70% of alleles in Italy), while other mutations have much lower frequency.
Valeria Tomati +23 more
doaj +1 more source