ArticleExpert review of precision medicine and drug development
Efficacy of lumacaftor-ivacaftor for the treatment of cystic fibrosis patients homozygous for the F508del-CFTR mutation.
Article in Expert review of precision medicine and drug development. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 27 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
27 citing papers in PubMed.
- Review
- Impact of lumacaftor/ivacaftor on nutrition and growth in modulator-naïve children over 24 weeks.Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2024Article
- Impaired intestinal free fatty acid transport followed by chylomicron malformation, not pancreatic insufficiency, cause metabolic defects in cystic fibrosis.Journal of lipid research · 2024Article
- Pyronaridine as a Bromodomain-Containing Protein 4-Molecules (Basel, Switzerland) · 2023Article
- Proximity Profiling of the CFTR Interaction Landscape in Response to Orkambi.International journal of molecular sciences · 2022Article
- Granulocytic Myeloid-Derived Suppressor Cells in Cystic Fibrosis.Frontiers in immunology · 2021Review
- Preclinical evaluation of the epithelial sodium channel inhibitor BI 1265162 for treatment of cystic fibrosis.ERJ open research · 2020Article
- Article
- Review
- Short-term consequences of F508del-CFTR thermal instability on CFTR-dependent transepithelial currents in human airway epithelial cells.Scientific reports · 2019Article
- Beyond cystic fibrosis transmembrane conductance regulator therapy: a perspective on gene therapy and small molecule treatment for cystic fibrosis.Gene therapy · 2019Review
- The effect of CFTR modulators on a cystic fibrosis patient presenting with recurrent pancreatitis in the absence of respiratory symptoms: a case report.BMC gastroenterology · 2019Article
- The cystic fibrosis airway milieu enhances rescue of F508del in a pre-clinical model.The European respiratory journal · 2018Article
- Chemically modified hCFTR mRNAs recuperate lung function in a mouse model of cystic fibrosis.Scientific reports · 2018Article
- Cysteine modifiers suggest an allosteric inhibitory site on the CAL PDZ domain.Bioscience reports · 2018Article
- High-Content Surface and Total Expression siRNA Kinase Library Screen with VX-809 Treatment Reveals Kinase Targets that Enhance F508del-CFTR Rescue.Molecular pharmaceutics · 2018Article
- Recent progress in translational cystic fibrosis research using precision medicine strategies.Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2018Review
- The EGFR-ADAM17 Axis in Chronic Obstructive Pulmonary Disease and Cystic Fibrosis Lung Pathology.Mediators of inflammation · 2018Review
- Cytochrome P450 3A4 Induction: Lumacaftor versus Ivacaftor Potentially Resulting in Significantly Reduced Plasma Concentration of Ivacaftor.Drug metabolism letters · 2018Article
- The potentially beneficial central nervous system activity profile of ivacaftor and its metabolites.ERJ open research · 2018Article
Corrections and comments
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Authors and funding
3 authors.
Funding
Abstract
Cystic fibrosis (CF) results from mutations in the CF transmembrane conductance regulator (CFTR) gene, which codes for the CFTR channel protein. The most common mutation in CF is F508del, which produces a misfolded protein with diminished channel activity. The development of small-molecule CFTR-modulator compounds offers an exciting and novel approach for pharmacological treatment of CF. The corrector lumacaftor helps rescue F508del-CFTR to the cell surface, and potentiator ivacaftor increases F508del-CFTR channel activity. The combination of lumacaftor-ivacaftor (Vertex Pharmaceuticals Incorporated) represents the first FDA-approved therapy for CF patients with two copies of the F508del mutation. Although this combination therapy is the first treatment to directly target the F508del-CFTR mutation, patients taking this drug displayed only modest improvements in lung function. This article summarizes recent data from clinical trials and research discoveries relating to the lumacaftor-ivacaftor treatment, and considers options for identifying future therapies that will be most efficacious for all CF patients.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.