Tezacaftor/Ivacaftor in Subjects with Cystic Fibrosis and F508del / F508del-CFTR or F508del / G551D-CFTR

Tezacaftor (formerly VX-661) is an investigational small molecule that improves processing and trafficking of the cystic fibrosis transmembrane conductance regulator (CFTR) in vitro, and improves CFTR function alone and in combination with ivacaftor. To evaluate the safety and efficacy of tezacaftor...

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Vydané v:	American journal of respiratory and critical care medicine Ročník 197; číslo 2; s. 214 - 224
Hlavní autori:	Donaldson, Scott H., Pilewski, Joseph M., Griese, Matthias, Cooke, Jon, Viswanathan, Lakshmi, Tullis, Elizabeth, Davies, Jane C., Lekstrom-Himes, Julie A., Wang, Linda T.
Médium:	Journal Article
Jazyk:	English
Vydavateľské údaje:	United States American Thoracic Society 15.01.2018
Predmet:	Administration, Oral Adolescent Adult Aminophenols - administration & dosage Aminophenols - adverse effects Benzodioxoles - administration & dosage Benzodioxoles - adverse effects Chloride Cystic fibrosis Cystic Fibrosis - diagnosis Cystic Fibrosis - drug therapy Cystic Fibrosis - genetics Cystic Fibrosis Transmembrane Conductance Regulator - genetics Cysts Dose-Response Relationship, Drug Double-Blind Method Drug Administration Schedule Drug dosages Drug Therapy, Combination Female Humans Indoles - administration & dosage Indoles - adverse effects Kinases Male Maximum Tolerated Dose Molecular Targeted Therapy - methods Mutation Original Patients Pediatrics Prognosis Proteins Quinolones - administration & dosage Quinolones - adverse effects Respiratory Function Tests Risk Assessment Severity of Illness Index Treatment Outcome Young Adult United States > US forced expiratory volume sweat chloride CFTR modulator cystic fibrosis transmembrane conductance regulator corrector
ISSN:	1073-449X, 1535-4970, 1535-4970
On-line prístup:	Získať plný text
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