lumacaftor (VX-809) / Vertex 
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 9 Diseases   1 Trial   1 Trial   672 News 


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  • ||||||||||  Pulmozyme (dornase alfa) / Roche
    Review, Journal:  Medical interventions for chronic rhinosinusitis in cystic fibrosis. (Pubmed Central) -  Oct 25, 2019   
    We identified no eligible trials assessing the medical interventions in people with cystic fibrosis and chronic rhinosinusitis. High-quality trials are needed which should assess the efficacy of different treatment options detailed above for managing chronic rhinosinusitis, preventing pulmonary exacerbations and improving quality of life in people with cystic fibrosis.
  • ||||||||||  lumacaftor (VX-809) / Vertex, Orkambi (lumacaftor/ivacaftor) / Vertex
    Review, Journal:  Lumacaftor/ivacaftor for cystic fibrosis. (Pubmed Central) -  Oct 22, 2019   
    High-quality trials are needed which should assess the efficacy of different treatment options detailed above for managing chronic rhinosinusitis, preventing pulmonary exacerbations and improving quality of life in people with cystic fibrosis. No abstract available
  • ||||||||||  lumacaftor (VX-809) / Vertex
    Journal:  A novel triple combination of pharmacological chaperones improves F508del-CFTR correction. (Pubmed Central) -  Oct 20, 2019   
    The triple combination of MCG1516A, RDR1, and VX-809 restored CFTR function to >20% that of non-CF cells in well differentiated HBE cells and to much higher levels in other cell types. Thus the results suggest the presence of at least three distinct sites for pharmacological chaperones on F508del-CFTR NBD1, encouraging the development of triple corrector combinations.
  • ||||||||||  Kalydeco (ivacaftor) / Vertex, lumacaftor (VX-809) / Vertex, Orkambi (lumacaftor/ivacaftor) / Vertex
    Journal:  A Proteomic Variant Approach (ProVarA) for Personalized Medicine of Inherited and Somatic Disease. (Pubmed Central) -  Oct 2, 2019   
    ProVarA represents the first comparative proteomic analysis among multiple disease-causing mutations, thereby providing a methodological approach that provides a significant advancement to existing proteomic efforts in understanding the impact of variation in CF disease. We posit that the implementation of ProVarA for any familial or somatic mutation will provide a substantial increase in the knowledge base needed to implement a precision medicine-based approach for clinical management of disease.
  • ||||||||||  lumacaftor (VX-809) / Vertex
    Journal:  Dissection of the Role of VIMP in Endoplasmic Reticulum-Associated Degradation of CFTRΔF508. (Pubmed Central) -  Sep 28, 2019   
    We further show that blocking CFTRΔF508 degradation by knockdown of VIMP substantially augments the effect of VX809, a drug that allows a fraction of CFTRΔF508 to fold properly and mobilize from ER to cell surface for normal functioning. This study provides insight into the role of VIMP in ERAD and presents a potential target for the treatment of cystic fibrosis patients carrying the CFTRΔF508 mutation.
  • ||||||||||  Kalydeco (ivacaftor) / Vertex, lumacaftor (VX-809) / Vertex
    Review, Journal:  Ion Channel Modulators in Cystic Fibrosis. (Pubmed Central) -  Sep 28, 2019   
    In this review, we focus on recent developments and remaining challenges of CFTR-directed therapies, as well as modulators of other ion channels such as alternative chloride channels and the epithelial sodium channel (ENaC) as additional targets in CF lung disease. Further, we discuss how patient-derived precision medicine models may aid the translation of emerging next generation ion channel modulators from the laboratory to the clinic and tailor their use for optimal therapeutic benefits in individual patients with CF.
  • ||||||||||  lumacaftor (VX-809) / Vertex
    Journal:  Guanosine monophosphate reductase 1 is a potential therapeutic target for Alzheimer's disease. (Pubmed Central) -  Sep 22, 2019   
    After 20 days of oral administration, we observed that β-Amyloid accumulation was slowed down, and phosphorylation of Tau was almost eliminated in the treated mice. We highlight the elevated expression level of GMPR in AD and propose a therapeutic strategy of inhibiting GMPR1 with lumacaftor.
  • ||||||||||  Cystic Fibrosis Patients Eligible for Modulator Drugs: Data from Cystic Fibrosis Registry of Turkey (TP-31) -  Aug 26, 2019 - Abstract #ERS2019ERS_4752;    
    Tezacaftor/Ivacaftor (T/I) is approved for people ages ≥12 who have two copies of the F508del mutation and also approved for people who have a single copy of one of 26 specific mutations...According to the data, approximately one fifth of CF patients are eligible for modulator drug therapy. Compared to North European and North American CF patients, less Turkish patients are eligible for modulator therapies, so new treatment modalities are necessary for them.
  • ||||||||||  lumacaftor (VX-809) / Vertex, Orkambi (lumacaftor/ivacaftor) / Vertex
    Pregnancy in two patients with cystic fibrosis treated with lumacaftor/ivacaftor (TP-31) -  Aug 26, 2019 - Abstract #ERS2019ERS_4751;    
    There might be an effect of CFTR-modulators on fecundity and the course and outcome of pregnancy in CF-patients. More data are needed to support the safety or risk for infants of mothers treated with CFTR-modulators.
  • ||||||||||  Kalydeco (ivacaftor) / Vertex, lumacaftor (VX-809) / Vertex, Orkambi (lumacaftor/ivacaftor) / Vertex
    The effect of CFTR modulators on CT outcomes in cystic fibrosis (8A) -  Aug 26, 2019 - Abstract #ERS2019ERS_2272;    
    Quantitative CT image biomarkers provide more sensitive assessments of treatment efficacy on lung structure and have the potential to be used in early interventional studies. However, a larger study population is needed to determine the long-term effects of therapy on structural lung disease.
  • ||||||||||  Kalydeco (ivacaftor) / Vertex, lumacaftor (VX-809) / Vertex, Orkambi (lumacaftor/ivacaftor) / Vertex
    Clinical, Journal:  Healthcare expenditures for privately insured US patients with cystic fibrosis, 2010-2016. (Pubmed Central) -  Aug 23, 2019   
    However, there was a high discontinuation rate due to adverse events. Per-patient expenditures for privately-insured patients with CF almost doubled during 2010-2016; specialty drugs were largely responsible for this increase, with a major contribution from new, genotype-targeted CFTR modulator medications.
  • ||||||||||  lumacaftor (VX-809) / Vertex
    Journal:  Guanylate cyclase 2C agonism corrects CFTR mutants. (Pubmed Central) -  Aug 1, 2019   
    GCC coexpression and activation facilitated processing and ER exit of F508del CFTR and presented a potentially novel rescue modality in the intestine, similar to the CF corrector VX-809. Our findings identify GCC as a biological CFTR corrector and potentiator in the intestine.
  • ||||||||||  lumacaftor (VX-809) / Vertex, Orkambi (lumacaftor/ivacaftor) / Vertex
    Biomarker, Journal:  Metabolomic responses to lumacaftor/ivacaftor in cystic fibrosis. (Pubmed Central) -  Jul 24, 2019   
    Selected key lipid and amino acid metabolic pathways were significantly affected by Lumacaftor/Ivacaftor initiation and similar pathways were affected in clinical responders. Targeted metabolomics may provide useful and relevant biomarkers of CFTR modulator responses.
  • ||||||||||  Kalydeco (ivacaftor) / Vertex, lumacaftor (VX-809) / Vertex
    Journal:  Nasospheroids permit measurements of CFTR-dependent fluid transport. (Pubmed Central) -  Jul 11, 2019   
    This model employs a simple measurement of size corresponding to changes in CFTR activity and is applicable for detection of small changes in CFTR activity from individual patients in vitro. Advancements of this technique will provide a robust model for individualized prediction of CFTR modulator efficacy.
  • ||||||||||  Kalydeco (ivacaftor) / Vertex, lumacaftor (VX-809) / Vertex, Orkambi (lumacaftor/ivacaftor) / Vertex
    Journal:  microRNA-145 Antagonism Reverses TGF-β Inhibition of F508del CFTR Correction in Airway Epithelia. (Pubmed Central) -  Jul 11, 2019   
    Specific antagonists to miR-145 interrupt TGF-β signaling to restore F508del CFTR modulation. miR-145 antagonism may offer a novel therapeutic opportunity to enhance therapeutic benefit of F508del CFTR correction in CF epithelia.
  • ||||||||||  Clinical, Journal:  Clinical development of triple-combination CFTR modulators for cystic fibrosis patients with one or two F508del alleles. (Pubmed Central) -  Jun 21, 2019   
    Next-generation CFTR correctors VX-659 and VX-445, each in triple combination with tezacaftor and ivacaftor, improve CFTR processing, trafficking and function in vitro and have demonstrated clinical improvements in phase 2 studies in patients with CF with one or two F508del-CFTR alleles. Here, we present the rationale and design of four randomised phase 3 studies, and their open-label extensions, evaluating VX-659 (ECLIPSE) or VX-445 (AURORA) plus tezacaftor and ivacaftor in patients with one or two F508del-CFTR alleles.
  • ||||||||||  lumacaftor (VX-809) / Vertex
    Clinical, Journal:  Characterization of Δ(G970-T1122)-CFTR, the most frequent CFTR mutant identified in Japanese cystic fibrosis patients. (Pubmed Central) -  May 30, 2019   
    ...However, VX-809 a CFTR corrector that can help maturation of ΔF508, had no effect on Δ(G970-T1122)...Japanese, and perhaps people in other Asian nations, carry a class II mutation Δ(G970-T1122) with a higher frequency than previously appreciated. Further study of the Δ(G970-T1122)-CFTR is essential for understanding CF and CFTR-related diseases particularly in Asian countries.
  • ||||||||||  Kalydeco (ivacaftor) / Vertex, lumacaftor (VX-809) / Vertex, Orkambi (lumacaftor/ivacaftor) / Vertex
    Clinical, Journal:  Cystic Fibrosis Pulmonary Guidelines: Use of CFTR Modulator Therapy in Patients with Cystic Fibrosis. (Pubmed Central) -  May 1, 2019   
    These recommendations will be of help to CF clinicians, patients, and their families in guiding decisions regarding use of these medications. We anticipate that these guidelines will be revised as newer data and medications become available.
  • ||||||||||  lumacaftor (VX-809) / Vertex
    Journal:  Identifying Inhibitors of the Hsp90-Aha1 Protein Complex, a Potential Target to Drug Cystic Fibrosis, by Alpha Technology. (Pubmed Central) -  Apr 19, 2019   
    We identified two druglike molecules that showed promising results when we tested their ability to restore chloride channel activity in culture cells expressing the mutant CFTRΔF508 protein. The two molecules were most effective in combination with the corrector VX-809 and may therefore serve as a lead compound that can be further developed into a drug to treat cystic fibrosis patients.
  • ||||||||||  Journal:  Tezacaftor for the treatment of cystic fibrosis. (Pubmed Central) -  Apr 5, 2019   
    The horizon for continued improvements in CFTR-targeted treatments is promising, with three-drug combinations currently in Phase 3 clinical trials, and other drugs with novel mechanisms of action being studied. Within the next 5 years, the vast majority of patients with CF are expected to have a modulator approved for their genotype.
  • ||||||||||  Kalydeco (ivacaftor) / Vertex, lumacaftor (VX-809) / Vertex, Orkambi (lumacaftor/ivacaftor) / Vertex
    Clinical, Journal:  Genetic and phenotypic traits of children and adolescents with cystic fibrosis in Southern Brazil. (Pubmed Central) -  Mar 22, 2019   
    More than half of the sample (52.38%) presented homozygous mutation for F508del, that is, patients who could be treated with Lumacaftor/Ivacaftor. Approximately 7% of the patients (7.14%) presented type III and IV mutations, therefore becoming candidates for the treatment with Ivacaftor.