British Journal of Pharmacology, Volume 182, Issue 24, Page 6063-6080, December 2025.Background and Purpose Cystic fibrosis (CF) is due to loss‐of‐function variants of the CF transmembrane conductance regulator (CFTR) channel. The most effective treatment for people with CF carrying the F508del mutation is the triple combination of elexacaftor–tezacaftor–ivacaftor (ETI).
Emanuela Pesce +25 more
wiley +1 more source
Are new models needed to optimize the utilization of new medicines to sustain healthcare systems? [PDF]
, 2014 Medicines have made an appreciable contribution to improving health. However, even high-income countries are struggling to fund new premium-priced medicines. This will grow necessitating the development of new models to optimize their use.
Acurcio, Francisco +39 more
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Consequences of cystic fibrosis transmembrane regulator mutations on inflammatory cells [PDF]
, 2016 Recent studies in cystic fibrosis (CF) transmembrane regulator (CFTR) mutations and function have shed light on its involvement in disease progression. The extent of cell and tissue distribution of CFTR facilitates systemic dysfunction of ion transport ...
Castro, Guillermo Raul +2 more
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A novel treatment of cystic fibrosis acting on-target:cysteamine plus epigallocatechin gallate for the autophagy-dependent rescue of class II-mutated CFTR [PDF]
, 2016 We previously reported that the combination of two safe proteostasis regulators, cysteamine and epigallocatechin gallate (EGCG), can be used to improve deficient expression of the cystic fibrosis transmembrane conductance regulator (CFTR) in patients ...
A Di Pasqua +68 more
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Understanding Pseudomonas status among adults with cystic fibrosis: a real-world comparison of the Leeds criteria against clinicians' decision [PDF]
, 2018 Pseudomonas aeruginosa status influences cystic fibrosis (CF) clinical management but no 'gold standard' definition exists. The Leeds criteria are commonly used but may lack sensitivity for chronic P. aeruginosa. We compared clinicians' decision with the
Allenby, M.I. +7 more
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Lumacaftor/ivacaftor in cystic fibrosis: effects on glucose metabolism and insulin secretion [PDF]
, 2021 Carla Colombo +9 more
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Mucociliary dysfunction in HIV and smoked substance abuse [PDF]
, 2015 Impaired mucociliary clearance (MCC) is a hallmark of acquired chronic airway diseases like chronic bronchitis associated with chronic obstructive pulmonary disease (COPD) and asthma.
Chinnapaiyan, Srinivasan +1 more
core +2 more sources
Personalised medicine of Cystic fibrosis [PDF]
, 2017 Treballs Finals de Grau de Farmàcia, Facultat de Farmàcia, Universitat de Barcelona, 2017. Tutor: Carlos Julián Ciudad i Gómez[en] The European commission defines personalised medicine as a medical approach that uses molecular insights into health and ...
Cabré Juan, Antoni
core
Small molecule anionophores promote transmembrane anion permeation matching CFTR activity [PDF]
, 2018 Anion selective ionophores, anionophores, are small molecules capable of facilitating the transmembrane transport of anions. Inspired in the structure of natural product prodigiosin, four novel anionophores 1a-d, including a 1,2,3-triazole group, were ...
A Fürstner +36 more
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