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The New England journal of medicine, ISSN 1533-4406, 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... 
Medicine, General & Internal | Life Sciences & Biomedicine | General & Internal Medicine | Science & Technology | Cystic Fibrosis - physiopathology | Humans | Pyrrolidines - administration & dosage | Pyrrolidines - adverse effects | Male | Chloride Channel Agonists - adverse effects | Benzodioxoles - administration & dosage | Aminophenols - adverse effects | Indoles - administration & dosage | Forced Expiratory Volume | Sweat - chemistry | Young Adult | Pyridines - adverse effects | Quinolones - adverse effects | Adult | Benzodioxoles - adverse effects | Female | Child | Pyrazoles - adverse effects | Pyridines - administration & dosage | Double-Blind Method | Genotype | Chlorides - analysis | Pyrazoles - administration & dosage | Quinolones - administration & dosage | Indoles - adverse effects | Chloride Channel Agonists - administration & dosage | Cystic Fibrosis - genetics | Adolescent | Cystic Fibrosis Transmembrane Conductance Regulator - genetics | Mutation | Aminophenols - administration & dosage | Cystic Fibrosis - drug therapy | Drug Combinations | 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 | Index Medicus | Abridged Index Medicus
Journal Article
The New England journal of medicine, ISSN 1533-4406, 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,... 
Medicine, General & Internal | Life Sciences & Biomedicine | General & Internal Medicine | Science & Technology | 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 | Cystic fibrosis transmembrane conductance regulator | Protein transport | Sweat | Binding sites | Genotypes | Index Medicus | Abridged Index Medicus
Journal Article
The New England journal of medicine, ISSN 1533-4406, 11/2017, Volume 377, Issue 21, pp. 2013 - 2023
Journal Article
The New England journal of medicine, ISSN 1533-4406, 11/2017, Volume 377, Issue 21, pp. 2024 - 2035
Journal Article
The New England journal of medicine, ISSN 1533-4406, 10/2018, Volume 379, Issue 17, pp. 1612 - 1620
... (VX-445-tezacaftor-ivacaftor). METHODS: We evaluated the effects of VX-445-tezacaftor-ivacaftor on Phe508del CFTR protein processing, trafficking, and chloride transport in human bronchial epithelial cells... 
Medicine, General & Internal | Life Sciences & Biomedicine | General & Internal Medicine | Science & Technology | 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 | Cystic fibrosis transmembrane conductance regulator | Protein transport | Sweat | Pharmaceuticals | Index Medicus | Abridged Index Medicus
Journal Article
The New England journal of medicine, ISSN 1533-4406, 07/2015, Volume 373, Issue 3, pp. 220 - 231
Journal Article
The Lancet (British edition), 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 | Life Sciences & Biomedicine | General & Internal Medicine | Science & Technology | 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 | Index Medicus | Abridged Index Medicus
Journal Article
Journal Article
American journal of respiratory and critical care medicine, ISSN 1535-4970, 01/2018, Volume 197, Issue 2, pp. 214 - 224
Journal Article