The New England journal of medicine, 2018-10, Vol.379 (17), p.1599-1611
- Davies, Jane C
- Moskowitz, Samuel M
- Brown, Cynthia
- Horsley, Alexander
- Mall, Marcus A
- McKone, Edward F
- Plant, Barry J
- Prais, Dario
- Ramsey, Bonnie W
- Taylor-Cousar, Jennifer L
- Tullis, Elizabeth
- Uluer, Ahmet
- McKee, Charlotte M
- Robertson, Sarah
- Shilling, Rebecca A
- Simard, Christopher
- Van Goor, Fredrick
- Waltz, David
- Xuan, Fengjuan
- Young, Tim
- Rowe, Steven M
2018
Details
- Autor(en) / Beteiligte
- Davies, Jane C
- Moskowitz, Samuel M
- Brown, Cynthia
- Horsley, Alexander
- Mall, Marcus A
- McKone, Edward F
- Plant, Barry J
- Prais, Dario
- Ramsey, Bonnie W
- Taylor-Cousar, Jennifer L
- Tullis, Elizabeth
- Uluer, Ahmet
- McKee, Charlotte M
- Robertson, Sarah
- Shilling, Rebecca A
- Simard, Christopher
- Van Goor, Fredrick
- Waltz, David
- Xuan, Fengjuan
- Young, Tim
- Rowe, Steven M
- Titel
- VX-659–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles
- Ist Teil von
- The New England journal of medicine, 2018-10, Vol.379 (17), p.1599-1611
- Ort / Verlag
- United States: Massachusetts Medical Society
- Erscheinungsjahr
- 2018
- Link zum Volltext
- Quelle
- Free E-Journal (出版社公開部分のみ）
- Beschreibungen/Notizen
- 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, sweat chloride concentration, and symptoms in patients with cystic fibrosis who harbored one or two Phe508del alleles.
- Sprache
- Englisch
- Identifikatoren
- ISSN: 0028-4793eISSN: 1533-4406DOI: 10.1056/NEJMoa1807119Titel-ID: cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_6277022
- Format
- –
- Schlagworte
- Adolescent, Adult, Alleles, Aminophenols - adverse effects, Aminophenols - therapeutic use, Benzodioxoles - adverse effects, Benzodioxoles - therapeutic use, Binding sites, Cells, Cultured, Chloride Channel Agonists - adverse effects, Chloride Channel Agonists - therapeutic use, Chloride transport, Chlorides - analysis, Clinical trials, Conductance, Cystic fibrosis, Cystic Fibrosis - drug therapy, Cystic Fibrosis - genetics, Cystic fibrosis transmembrane conductance regulator, Cystic Fibrosis Transmembrane Conductance Regulator - genetics, Cystic Fibrosis Transmembrane Conductance Regulator - metabolism, Dosage and administration, Double-Blind Method, Drug Combinations, Drug therapy, Epithelial cells, Female, Forced Expiratory Volume - drug effects, Genetic aspects, Genotype, Genotype & phenotype, Genotypes, Humans, Indoles - adverse effects, Indoles - therapeutic use, Ivacaftor, Male, Mutation, Patients, Protein transport, Proteins, Pyrazoles - adverse effects, Pyrazoles - pharmacology, Pyrazoles - therapeutic use, Pyrrolidines - adverse effects, Pyrrolidines - pharmacology, Pyrrolidines - therapeutic use, Quinolones - adverse effects, Quinolones - therapeutic use, Recovery of function, Sweat, Sweat - chemistry, Young Adult