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The New England Journal of Medicine, ISSN 0028-4793, 10/2018, Volume 379, Issue 17, pp. 1599 - 1611
This companion article to the VX-445 report shows that VX-659, a new CFTR potentiator, when administered with tezacaftor and ivacaftor improved lung function,... 
G551D MUTATION | MEDICINE, GENERAL & INTERNAL | IN-VITRO | CFTR POTENTIATOR | TEZACAFTOR-IVACAFTOR | Pyrazoles - therapeutic use | Forced Expiratory Volume - drug effects | Humans | Pyrrolidines - adverse effects | Chloride Channel Agonists - therapeutic use | Male | Aminophenols - therapeutic use | Chloride Channel Agonists - adverse effects | Aminophenols - adverse effects | Pyrrolidines - pharmacology | Pyrrolidines - therapeutic use | Sweat - chemistry | Young Adult | Quinolones - adverse effects | Quinolones - therapeutic use | Adult | Benzodioxoles - adverse effects | Female | Benzodioxoles - therapeutic use | Pyrazoles - adverse effects | Pyrazoles - pharmacology | Double-Blind Method | Cells, Cultured | Genotype | Cystic Fibrosis Transmembrane Conductance Regulator - metabolism | Chlorides - analysis | Indoles - adverse effects | Cystic Fibrosis - genetics | Adolescent | Alleles | Cystic Fibrosis Transmembrane Conductance Regulator - genetics | Indoles - therapeutic use | Mutation | Cystic Fibrosis - drug therapy | Drug Combinations | Cystic fibrosis | Ivacaftor | Genetic aspects | Dosage and administration | Drug therapy | Epithelial cells | Chloride transport | Clinical trials | Recovery of function | Patients | Proteins | Genotype & phenotype | Conductance | Full text | Cystic fibrosis transmembrane conductance regulator | Protein transport | Sweat | Binding sites | Genotypes
Journal Article
The New England Journal of Medicine, ISSN 0028-4793, 07/2015, Volume 373, Issue 3, pp. 220 - 231
Journal Article
The New England Journal of Medicine, ISSN 0028-4793, 11/2017, Volume 377, Issue 21, pp. 2013 - 2023
Journal Article
The New England Journal of Medicine, ISSN 0028-4793, 11/2017, Volume 377, Issue 21, pp. 2024 - 2035
Journal Article
The New England Journal of Medicine, ISSN 0028-4793, 10/2018, Volume 379, Issue 17, pp. 1612 - 1620
This preclinical, phase 2 report shows that VX-445, a CFTR potentiator when administered with tezacaftor and ivacaftor, improved lung function and reduced... 
G551D MUTATION | MEDICINE, GENERAL & INTERNAL | TEZACAFTOR-IVACAFTOR | CFTR | Pyrazoles - therapeutic use | Forced Expiratory Volume - drug effects | Humans | Pyrrolidines - administration & dosage | Chloride Channel Agonists - therapeutic use | Male | Aminophenols - therapeutic use | Chloride Channel Agonists - adverse effects | Aminophenols - adverse effects | Pyrrolidines - pharmacology | Pyrrolidines - therapeutic use | Sweat - chemistry | Young Adult | Quinolones - adverse effects | Chlorides - metabolism | Quinolones - therapeutic use | Adult | Benzodioxoles - adverse effects | Female | Benzodioxoles - therapeutic use | Pyrazoles - pharmacology | Pyridines - therapeutic use | Pyridines - administration & dosage | Double-Blind Method | Genotype | Cystic Fibrosis Transmembrane Conductance Regulator - metabolism | Chlorides - analysis | Pyrazoles - administration & dosage | Indoles - adverse effects | Cystic Fibrosis - genetics | Adolescent | Alleles | Cystic Fibrosis Transmembrane Conductance Regulator - genetics | Indoles - therapeutic use | Pyridines - pharmacology | Mutation | Cystic Fibrosis - drug therapy | Drug Combinations | Cystic fibrosis | Ivacaftor | Dosage and administration | Drug therapy | Statistical analysis | Writers | Epithelial cells | Chloride transport | Recovery of function | Patients | Design | Proteins | Genotype & phenotype | Chloride | Collaboration | Conductance | Full text | Cystic fibrosis transmembrane conductance regulator | Protein transport | Sweat | Pharmaceuticals
Journal Article
Journal Article
The New England Journal of Medicine, ISSN 0028-4793, 11/2010, Volume 363, Issue 21, pp. 1991 - 2003
Journal Article
Contact Dermatitis, ISSN 0105-1873, 04/2014, Volume 70, Issue 4, pp. 233 - 237
Journal Article
The New England Journal of Medicine, ISSN 0028-4793, 11/2011, Volume 365, Issue 18, pp. 1663 - 1672
Journal Article
Journal of Cystic Fibrosis, ISSN 1569-1993, 2017, Volume 16, Issue 3, pp. 392 - 394
Abstract Safety-data for lumacaftor/ivacaftor (LUM/IVA) combination therapy in patients with severe lung disease (percent predicted forced expiratory volume in... 
Pulmonary/Respiratory | Cystic fibrosis | Lung function, adverse events | Ivacaftor | Lumacaftor | Lung function | RESPIRATORY SYSTEM | COMBINATION THERAPY | PHE508DEL CFTR | adverse events | Forced Expiratory Volume - drug effects | Cystic Fibrosis - physiopathology | Humans | Aminopyridines - adverse effects | Male | Dyspnea - etiology | Drug Monitoring - methods | Benzodioxoles - administration & dosage | Aminophenols - adverse effects | Quinolones - adverse effects | Adult | Benzodioxoles - adverse effects | Female | Severity of Illness Index | Aminopyridines - administration & dosage | Dyspnea - diagnosis | Respiratory Function Tests - methods | Treatment Outcome | Membrane Transport Modulators - administration & dosage | Quinolones - administration & dosage | Cystic Fibrosis - genetics | Cystic Fibrosis - diagnosis | Cystic Fibrosis Transmembrane Conductance Regulator - genetics | Mutation | Australia | Membrane Transport Modulators - adverse effects | Drug-Related Side Effects and Adverse Reactions - physiopathology | Aminophenols - administration & dosage | Cystic Fibrosis - drug therapy | Drug Combinations | Drug-Related Side Effects and Adverse Reactions - diagnosis | Medicine, Experimental | Complications and side effects | Medical research | Medical colleges | Pharmacy | Drugstores | Respiratory agents
Journal Article
American Journal of Respiratory and Critical Care Medicine, ISSN 1073-449X, 01/2018, Volume 197, Issue 2, pp. 214 - 224
Rationale: Tezacaftor (formerly VX-661) is an investigational small molecule that improves processing and trafficking of the cystic fibrosis transmembrane... 
Sweat chloride | Cystic fibrosis transmembrane conductance regulator corrector | Forced expiratory volume | CFTR modulator | EFFICACY | SAFETY | LUMACAFTOR-IVACAFTOR | IDENTIFICATION | INVESTIGATIONAL CFTR CORRECTOR | TRANSMEMBRANE CONDUCTANCE REGULATOR | G551D MUTATION | F508DEL-CFTR MUTATION | GENE | RESPIRATORY SYSTEM | cystic fibrosis transmembrane conductance regulator corrector | PHE508DEL CFTR | forced expiratory volume | sweat chloride | CRITICAL CARE MEDICINE | Prognosis | Humans | Male | Benzodioxoles - administration & dosage | Aminophenols - adverse effects | Indoles - administration & dosage | Dose-Response Relationship, Drug | Young Adult | Quinolones - adverse effects | Adult | Benzodioxoles - adverse effects | Female | Drug Therapy, Combination | Molecular Targeted Therapy - methods | Severity of Illness Index | Double-Blind Method | Drug Administration Schedule | Risk Assessment | Administration, Oral | Treatment Outcome | Maximum Tolerated Dose | Quinolones - administration & dosage | Indoles - adverse effects | Cystic Fibrosis - genetics | Adolescent | Cystic Fibrosis - diagnosis | Cystic Fibrosis Transmembrane Conductance Regulator - genetics | Respiratory Function Tests | Aminophenols - administration & dosage | Cystic Fibrosis - drug therapy | Proteins | Pediatrics | Chloride | Cysts | Cystic fibrosis | Mutation | Kinases | Patients | Drug dosages | Original
Journal Article