학술논문
Efficacy and safety of lumacaftor and ivacaftor in patients aged 6–11 years with cystic fibrosis homozygous for F508del-CFTR: a randomised, placebo-controlled phase 3 trial
Document Type
Article
Author
Ratjen, Felix; Hug, Christopher; Marigowda, Gautham; Tian, Simon; Huang, Xiaohong; Stanojevic, Sanja; Milla, Carlos E; Robinson, Paul D; Waltz, David; Davies, Jane C; Rosenfeld, Margaret; Starner, Timothy; Retsch-Bogart, George; Chmiel, James; Orenstein, David; Milla, Carlos; Rubenstein, Ronald; Walker, Seth; Cornell, Alexandra; Asfour, Fadi; Black, Philip; Colombo, John; Froh, Deborah; McColley, Susanna; Ruiz, Fadel; Quintero, Diana; Casey, Alicia; Mueller, Gary; Flume, Patrick; Livingston, Floyd; Rock, Michael; O'Sullivan, Brian; Schmidt, Howard; Lahiri, Thomas; McNamara, John; Chidekel, Aaron; Sass, Laura; Keens, Thomas; Schaeffer, David; Solomon, Melinda; Chilvers, Mark; Lands, Larry; Junge, Sibylle; Griese, Matthias; Staab, Doris; Pressler, Tacjana; van Koningsburggen-Rietschel, Silke; Naehrlich, Lutz; Reid, Alastair; Balfour-Lynn, Ian; Urquhart, Don; Lee, Timothy; Munck, Anne; Gaudelus, Isabelle Sermet; De Boeck, Christiane; Reix, Philippe; Malfroot, Anne; Bui, Stéphanie; Selvadurai, Hiranjan; Robinson, Philip; Wainwright, Claire; Clements, Barry; Hilton, Jodi; Hjelte, Lena
Source
The Lancet Respiratory Medicine; July 2017, Vol. 5 Issue: 7 p557-567, 11p
Subject
Language
ISSN
22132600; 22132619
Abstract
Lumacaftor and ivacaftor combination treatment showed efficacy in patients aged 12 years or older with cystic fibrosis homozygous for F508del-cystic fibrosis transmembrane conductance regulator (CFTR)in placebo-controlled studies and patients aged 6–11 years with cystic fibrosis homozygous for F508del-CFTRin an open-label study. We report efficacy and safety of lumacaftor and ivacaftor in patients with cystic fibrosis aged 6–11 years homozygous for F508del-CFTR.