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

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

Publisher

Elsevier BV

Subject

Pulmonary and Respiratory Medicine

Reference38 articles.

1. Cystic fibrosis;O'Sullivan;Lancet,2009

2. Identification and regulation of the cystic fibrosis transmembrane conductance regulator-generated chloride channel;Berger;J Clin Invest,1991

3. Aberrant CFTR-dependent HCO3- transport in mutations associated with cystic fibrosis;Choi;Nature,2001

4. Targeting a genetic defect: cystic fibrosis transmembrane conductance regulator modulators in cystic fibrosis;Derichs;Eur Respir Rev,2013

5. Clinical and Functional Translation of CFTR (CFTR2), https://www.cftr2.org/index.php (accessed Feb 15, 2017).

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