Efficacy and safety of ivacaftor in patients aged 6 to 11 years with cystic fibrosis with a G551D mutation

American Journal of Respiratory and Critical Care Medicine

Volumn 187, Issue 11, 2013, Pages 1219-1225

  Davies, Jane C ^a,b   Wainwright, Claire E ^c   Canny, Gerard J ^d   Chilvers, Mark A ^e   Howenstine, Michelle S ^f   Munck, Anne ^g   Mainz, Jochen G ^h   Rodriguez, Sally ⁱ   Li, Haihong ⁱ   Yen, Karl ⁱ   Ordoñez, Claudia L ⁱ   Ahrens, Richard ^j  

^a ROYAL BROMPTON AND HAREFIELD NHS FOUNDATION TRUST   (United Kingdom)

^b IMPERIAL COLLEGE LONDON   (United Kingdom)

^c UNIVERSITY OF QUEENSLAND   (Australia)

^d OUR LADY S CHILDREN S HOSPITAL   (Ireland)

^e BC CHILDREN S HOSPITAL   (Canada)

^f RILEY HOSPITAL FOR CHILDREN   (United States)

^g HÔPITAL ROBERT DEBRÉ   (France)

^h JENA UNIVERSITY HOSPITAL   (Germany)

ⁱ VERTEX PHARMACEUTICALS   (United States)

^j UNIVERSITY OF IOWA   (United States)

more..

Author keywords

Cystic fibrosis; Cystic fibrosis transmembrane conductance regulator protein; Cystic fibrosis, pulmonary; G551D CFTR mutation; Sweat test

Indexed keywords

CHLORIDE; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; IVACAFTOR; PLACEBO;

ADD ON THERAPY; ARTICLE; BODY WEIGHT; CHILD; CLINICAL ARTICLE; CLINICAL EVALUATION; CYSTIC FIBROSIS; DOUBLE BLIND PROCEDURE; DRUG EFFECT; DRUG EFFICACY; DRUG SAFETY; DRUG WITHDRAWAL; ENZYME ACTIVITY; FEMALE; FORCED EXPIRATORY VOLUME; HUMAN; INCIDENCE; LUNG FUNCTION; MALE; PREDICTION; PRESCHOOL CHILD; PRESCRIPTION; PRIORITY JOURNAL; RANDOMIZED CONTROLLED TRIAL; SCHOOL CHILD; SWEAT; TREATMENT DURATION; UNSPECIFIED SIDE EFFECT;

ADMINISTRATION, ORAL; ALLELES; AMINOPHENOLS; CHILD; CYSTIC FIBROSIS; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; DNA; DOSE-RESPONSE RELATIONSHIP, DRUG; DOUBLE-BLIND METHOD; FORCED EXPIRATORY VOLUME; HUMANS; LUNG; MUTATION; QUINOLONES; RESPIRATORY FUNCTION TESTS; TREATMENT OUTCOME;

EID: 84878970875     PISSN: 1073449X     EISSN: 15354970     Source Type: Journal    
DOI: 10.1164/rccm.201301-0153OC     Document Type: Article

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