Effect of ivacaftor in patients with advanced cystic fibrosis and a G551D-CFTR mutation: Safety and efficacy in an expanded access program in the United States

Journal of Cystic Fibrosis

Volumn 15, Issue 1, 2016, Pages 116-122

  Taylor Cousar, Jennifer ^a   Niknian, Minoo ^b   Gilmartin, Geoffrey ^b   Pilewski, Joseph M ^c  

^a DEPARTMENT OF MEDICINE   (United States)

^b VERTEX PHARMACEUTICALS   (United States)

^c UNIVERSITY OF PITTSBURGH   (United States)

Author keywords

Cystic fibrosis (CF); Cystic fibrosis transmembrane conductance regulator (CFTR); Lung function

Indexed keywords

IVACAFTOR; AMINOPHENOL DERIVATIVE; CFTR PROTEIN, HUMAN; CHLORIDE CHANNEL STIMULATING AGENT; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; QUINOLONE DERIVATIVE;

ABDOMINAL PAIN; ADOLESCENT; ADRENAL CORTEX INSUFFICIENCY; ADULT; ADVANCED CYSTIC FIBROSIS; ARTICLE; CHILD; CLINICAL ARTICLE; COUGHING; CYSTIC FIBROSIS; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR GENE; DISEASE SEVERITY; DIZZINESS; DRUG EFFICACY; DRUG SAFETY; DRUG WITHDRAWAL; DYSPNEA; FAINTNESS; FEMALE; FORCED EXPIRATORY VOLUME; G551D GENE; GASTROENTERITIS; GENE; GENE MUTATION; HEADACHE; HEMOPTYSIS; HUMAN; LIVER DYSFUNCTION; LUNG DISEASE; LUNG FUNCTION; LUNG TRANSPLANTATION; MALE; OPEN STUDY; PNEUMOTHORAX; RASH; RESPIRATORY FAILURE; SIDE EFFECT; SPUTUM; UNITED STATES; UPPER RESPIRATORY TRACT INFECTION; WEIGHT; WEIGHT GAIN; ANIMAL; CLINICAL TRIAL; DOSE RESPONSE; DRUG MONITORING; GENETICS; LUNG FUNCTION TEST; MUTATION; PROCEDURES; TREATMENT OUTCOME;

ADOLESCENT; ADULT; AMINOPHENOLS; ANIMALS; CHILD; CHLORIDE CHANNEL AGONISTS; CYSTIC FIBROSIS; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; DOSE-RESPONSE RELATIONSHIP, DRUG; DRUG MONITORING; FEMALE; HUMANS; MALE; MUTATION; QUINOLONES; RESPIRATORY FUNCTION TESTS; TREATMENT OUTCOME;

EID: 84959544874     PISSN: 15691993     EISSN: 18735010     Source Type: Journal    
DOI: 10.1016/j.jcf.2015.01.008     Document Type: Article

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