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The New England Journal of Medicine, ISSN 0028-4793, 11/2019, Volume 381, Issue 19, pp. 1809 - 1819
Triple treatment with elexacaftor, tezacaftor, and ivacaftor in patients with cystic fibrosis who had one Phe508del allele and a minimal-function mutation... 
Chlorides/analysis | Double-Blind Method | Humans | Pyridines/administration & dosage | Genotype | Male | Pyrazoles/administration & dosage | Forced Expiratory Volume | Indoles/administration & dosage | Young Adult | Aminophenols/administration & dosage | Cystic Fibrosis Transmembrane Conductance Regulator/genetics | Cystic Fibrosis/drug therapy | Quinolones/administration & dosage | Adolescent | Sweat/chemistry | Adult | Female | Mutation | Benzodioxoles/administration & dosage | Chloride Channel Agonists/administration & dosage | Child | Pyrrolidines/administration & dosage | Drug Combinations | TRANSMEMBRANE CONDUCTANCE REGULATOR | ADULT PATIENTS | MEDICINE, GENERAL & INTERNAL | EXACERBATIONS | FEV1 | POTENTIATOR | DECLINE | Cystic Fibrosis - physiopathology | Pyrrolidines - administration & dosage | Pyrrolidines - adverse effects | Chloride Channel Agonists - adverse effects | Benzodioxoles - administration & dosage | Aminophenols - adverse effects | Indoles - administration & dosage | Sweat - chemistry | Pyridines - adverse effects | Quinolones - adverse effects | Benzodioxoles - adverse effects | Pyrazoles - adverse effects | Pyridines - administration & dosage | Chlorides - analysis | Pyrazoles - administration & dosage | Quinolones - administration & dosage | Indoles - adverse effects | Chloride Channel Agonists - administration & dosage | Cystic Fibrosis - genetics | Cystic Fibrosis Transmembrane Conductance Regulator - genetics | Aminophenols - administration & dosage | Cystic Fibrosis - drug therapy | Cystic fibrosis | Ivacaftor | Genetic aspects | Drug therapy | Patient outcomes | Statistical analysis | Stock options | Clinical trials | Patients | Clinical outcomes | Quality of life | Proteins | Collaboration | Conductance | Genotypes | Pharmaceuticals
Journal Article
The New England Journal of Medicine, ISSN 0028-4793, 07/2015, Volume 373, Issue 3, pp. 220 - 231
Journal Article
The Lancet, ISSN 0140-6736, 11/2019, Volume 394, Issue 10212, pp. 1940 - 1948
Cystic fibrosis transmembrane conductance regulator (CFTR) modulators correct the basic defect caused by CFTR mutations. Improvements in health outcomes have... 
Double-Blind Method | Humans | yes | Pyridines/administration & dosage | Male | Pyrazoles/administration & dosage | Indoles/administration & dosage | Aminophenols/administration & dosage | Cystic Fibrosis Transmembrane Conductance Regulator/genetics | Cystic Fibrosis/drug therapy | Quinolones/administration & dosage | Adolescent | Sweat/chemistry | Female | Benzodioxoles/administration & dosage | Chloride Channel Agonists/administration & dosage | Drug Therapy, Combination | Child | Pyrrolidines/administration & dosage | MEDICINE, GENERAL & INTERNAL | GUIDELINES | Pyrrolidines - administration & dosage | Pyrrolidines - adverse effects | Chloride Channel Agonists - adverse effects | Benzodioxoles - administration & dosage | Aminophenols - adverse effects | Indoles - administration & dosage | Sweat - chemistry | Pyridines - adverse effects | Quinolones - adverse effects | Benzodioxoles - adverse effects | Pyrazoles - adverse effects | Pyridines - administration & dosage | Pyrazoles - administration & dosage | Quinolones - administration & dosage | Indoles - adverse effects | Chloride Channel Agonists - administration & dosage | Cystic Fibrosis - genetics | Cystic Fibrosis Transmembrane Conductance Regulator - genetics | Aminophenols - administration & dosage | Cystic Fibrosis - drug therapy | Clinical trials | Cystic fibrosis | Ivacaftor | Genetic aspects | Infections | Modulators | Defects | Quality of life | Resistance | Proteins | Randomization | Chloride | Chlorides | Fibrosis | Conductance | Mutation | Safety | Sweat | Active control
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
Lancet Respiratory Medicine, The, ISSN 2213-2600, 2017, Volume 5, Issue 7, pp. 557 - 567
Journal Article
Lancet Respiratory Medicine, The, ISSN 2213-2600, 2016, Volume 5, Issue 2, pp. 107 - 118
Journal Article
Science Translational Medicine, ISSN 1946-6234, 2019, Volume 11, Issue 485, p. eaau7531
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
PLoS ONE, ISSN 1932-6203, 04/2017, Volume 12, Issue 4, p. e0175486
We optically measured effects of orally available ivacaftor (Kalydeco (R)) on sweat rates of identified glands in 3 R117H subjects, each having a unique set of... 
TRANSMEMBRANE CONDUCTANCE REGULATOR | GLANDS | GENE | MESSENGER-RNA TRANSCRIPTS | PARTIAL PENETRANCE | MULTIDISCIPLINARY SCIENCES | CODING SEQUENCES | MUTATION | DISEASE | CYSTIC-FIBROSIS | CFTR POTENTIATOR | Muscarinic Agonists - pharmacology | Sweat - secretion | Humans | Male | Quinolones - pharmacology | Aminophenols - pharmacology | Methacholine Chloride - administration & dosage | Gene Deletion | Methacholine Chloride - pharmacology | Adrenergic beta-Agonists - administration & dosage | Adult | Female | Injections, Intradermal | Muscarinic Agonists - administration & dosage | Administration, Oral | Chloride Channel Agonists - pharmacology | Adrenergic beta-Agonists - pharmacology | Sweating - genetics | Exons - genetics | Cystic Fibrosis Transmembrane Conductance Regulator - metabolism | Sweating - drug effects | Quinolones - administration & dosage | Ion Channel Gating - genetics | Analysis of Variance | Chloride Channel Agonists - administration & dosage | Cystic Fibrosis Transmembrane Conductance Regulator - genetics | Mutation | Aminophenols - administration & dosage | Ion Channel Gating - drug effects | Ivacaftor | Care and treatment | Gene mutations | Methacholine | Cystic fibrosis | Dosage and administration | Research | Wine | Pediatrics | Acetylcholine receptors (muscarinic) | Laboratories | Linkage disequilibrium | Glands | Biology | Iontophoresis | Membrane conductance | Chloride | Intestine | Anion channels | Genetics | Physiology | Children | Sweat | Digestive tract | Gating | Channel opening | Secretion | Channel gating | Bioassays | Organs | Chloride transport | Ion transport | Medical screening | Medicine | Clear cells | Lungs | Alleles | In vivo methods and tests | Conductance | Cystic fibrosis transmembrane conductance regulator | Molecular biology
Journal Article