Frontiers in Pharmacology, 2021 Objectives: Cystic fibrosis transmembrane conductance regulator (CFTR) modulators, Kalydeco® (ivacaftor), Orkambi® (lumacaftor/ivacaftor) and Symkevi® (tezacaftor/ivacaftor), have substantially improved patients’ lives yet significantly burden healthcare
Khadidja Abdallah +3 more
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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
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In Silico Screening of Novel TMPRSS2 Inhibitors for Treatment of COVID-19
Molecules, 2022 COVID-19, a pandemic caused by the virus SARS-CoV-2, has spread globally, necessitating the search for antiviral compounds. Transmembrane protease serine 2 (TMPRSS2) is a cell surface protease that plays an essential role in SARS-CoV-2 infection ...
Shuo Wang, Xuexun Fang, Ye Wang
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Nationwide lung function monitoring from infancy in newborn-screened children with cystic fibrosis
ERJ Open Research, 2023 Background Cystic fibrosis (CF) lung disease starts in infancy and can be assessed for structural lung abnormalities using computed tomography or magnetic resonance scans, or for lung function impairment using multiple breath washout (MBW).
Rikke M. Sandvik +12 more
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tert-Butyl 3-(3-methyl-1-oxidopyridin-1-ium-2-yl)benzoate
IUCrData, 2016 In the title compound, C17H19NO3, which was obtained by oxidation of the corresponding pyridine derivative, the dihedral angle between the benzene and the pyridine rings is 68.2 (1)°.
Gerhard Laus +4 more
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Experience of using lumacaftor/ivacaftor in children with cystic fibrosis in the Astrakhan region
Acta Biomedica Scientifica, 2022 Background. Targeted therapy in patients with cystic fibrosis, which aims to restore the function of the cystic fibrosis transmembrane conductance regulator protein, is currently revolutionary in the treatment of the disease.
A. R. Kosareva +2 more
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Innovative Therapeutic Strategies for Cystic Fibrosis: Moving Forward to CRISPR Technique
Frontiers in Pharmacology, 2018 One of the most revolutionary technologies in recent years in the field of molecular biology is CRISPR-Cas9. CRISPR technology is a promising tool for gene editing that provides researchers the opportunity to easily alter DNA sequences and modify gene ...
Michele Marangi +2 more
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Long-term impact of Elexacaftor/Tezacaftor/ivacaftor on pulmonary, nutritional and metabolic outcomes in homozygous F508del cystic fibrosis patients: A real-world cohort study. [PDF]
Br J Clin PharmacolAim Elexacaftor/tezacaftor/ivacaftor (ETI) has markedly improved cystic fibrosis (CF) outcomes. However, its long‐term impact on nutrition, metabolism and liver health remains underexplored. We assessed 30‐month changes in pulmonary, nutritional, metabolic and inflammatory markers in people with CF (PwCF) homozygous for F508del.
Perrotta N +5 more
europepmc +2 more sources
Variable Responses to CFTR Correctors in vitro: Estimating the Design Effect in Precision Medicine
Frontiers in Pharmacology, 2018 Interest in precision medicine has grown in recent years due to the variable clinical benefit provided by some medications, their cost, and by new opportunities to tailor therapies to individual patients.
Elizabeth Matthes +14 more
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Italian Journal of Pediatrics, 2021 Background Cystic Fibrosis Related Diabetes (CFRD) is a frequent comorbidity of patients with Cystic Fibrosis (CF). A worsening of clinical conditions appears before CFRD.
Francesco Maria Rosanio +7 more
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