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
doaj +1 more source
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
doaj +1 more source
Modulator Therapy for Cystic Fibrosis: An Exploration of Current Research [PDF]
, 2020 Developing a drug therapy that addresses the root cause of cystic fibrosis (CF) by increasing CFTR protein levels has long been a research challenge. After genetic therapy failed because a suitable delivery system could not be found, researchers began ...
Rombocos, Jessalyn
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Two Small Molecules Restore Stability to a Sub-population of the Cystic Fibrosis Transmembrane conductance Regulator with the Predominant Disease-causing Mutation [PDF]
, 2017 Cystic fibrosis (CF) is caused by mutations that disrupt the plasma membraneexpression, stability, and function of the cystic fibrosis transmembrane conductance regulator (CFTR) Cl- channel.
Cai, Zhiwei +8 more
core +2 more sources
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
doaj +1 more source
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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From CFTR biology toward combinatorial pharmacotherapy:expanded classification of cystic fibrosis mutations [PDF]
, 2016 More than 2000 mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) have been described that confer a range of molecular cell biological and functional phenotypes.
Avramescu, Radu G. +17 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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International Committee on Mental Health in Cystic Fibrosis: Cystic Fibrosis Foundation and European Cystic Fibrosis Society consensus statements for screening and treating depression and anxiety [PDF]
, 2015 Studies measuring psychological distress in individuals with cystic fibrosis (CF) have found high rates of both depression and anxiety. Psychological symptoms in both individuals with CF and parent caregivers have been associated with decreased lung ...
Abbott, J +8 more
core +1 more source
Lumacaftor/ivacaftor changes the lung microbiome and metabolome in cystic fibrosis patients
ERJ Open Research, 2021 Rationale Targeted cystic fibrosis (CF) therapy with lumacaftor/ivacaftor partly restores chloride channel function and improves epithelial fluid transport in the airways.
Anne H. Neerincx +9 more
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