A CFTR corrector (lumacaftor) and a CFTR potentiator (ivacaftor) for treatment of patients with cystic fibrosis who have a phe508del CFTR mutation: A phase 2 randomised controlled trial

The Lancet Respiratory Medicine

Volumn 2, Issue 7, 2014, Pages 527-538

  Boyle, Michael P ^a   Bell, Scott C ^b   Konstan, Michael W ^c   McColley, Susanna A ^d   Rowe, Steven M ^e   Rietschel, Ernst ^f   Huang, Xiaohong ^g   Waltz, David ^g   Patel, Naimish R ^h   Rodman, David ⁱ  

^a DEPARTMENT OF PATHOLOGY   (United States)

^b THE PRINCE CHARLES HOSPITAL   (Australia)

^c CASE WESTERN RESERVE UNIVERSITY SCHOOL OF MEDICINE   (United States)

^d NORTHWESTERN UNIVERSITY   (United States)

^e UNIVERSITY OF ALABAMA AT BIRMINGHAM   (United States)

^f UNIVERSITY OF COLOGNE   (Germany)

^g VERTEX PHARMACEUTICALS   (United States)

^h ASTRAZENECA   (United Kingdom)

ⁱ MiRagen Therapeutics   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

CHLORIDE; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; IVACAFTOR; LUMACAFTOR; PHENYLALANINE; PLACEBO; 1,3 BENZODIOXOLE DERIVATIVE; AMINOPHENOL DERIVATIVE; AMINOPYRIDINE DERIVATIVE; CFTR PROTEIN, HUMAN; QUINOLONE DERIVATIVE;

ADULT; AGE; ARTICLE; AUSTRALIA; BELGIUM; BREATHING DISORDER; CFTR GENE; CHEST TIGHTNESS; COHORT ANALYSIS; CONTROLLED STUDY; COUGHING; CYSTIC FIBROSIS; DECREASED APPETITE; DIARRHEA; DISEASE EXACERBATION; DOUBLE BLIND PROCEDURE; DRUG ERUPTION; DRUG FEVER; DRUG HYPERSENSITIVITY; DRUG INDUCED HEADACHE; DRUG MEGADOSE; DRUG SAFETY; DRUG WITHDRAWAL; DYSPNEA; ELECTROLYTE DISTURBANCE; FEMALE; FORCED EXPIRATORY VOLUME; GENE DELETION; GENE MUTATION; GERMANY; HEMOPTYSIS; HETEROZYGOTE; HOMOZYGOTE; HUMAN; LUNG DISEASE; LUNG INSUFFICIENCY; MAJOR CLINICAL STUDY; MALE; MONOTHERAPY; MULTICENTER STUDY; NAUSEA; NEW ZEALAND; NOSE OBSTRUCTION; OROPHARYNX PAIN; OUTCOME ASSESSMENT; PATIENT COMPLIANCE; PHASE 2 CLINICAL TRIAL; PRACTICE GUIDELINE; PREDICTION; PRIORITY JOURNAL; RANDOMIZED CONTROLLED TRIAL; RESPIRATORY TRACT DISEASE; SINUS CONGESTION; SWEAT; TREATMENT DURATION; TREATMENT RESPONSE; UNITED STATES; UPPER RESPIRATORY TRACT INFECTION; ADOLESCENT; CHEMISTRY; CLINICAL TRIAL; COMBINATION DRUG THERAPY; GENETICS; NUCLEOTIDE SEQUENCE; PATHOPHYSIOLOGY; YOUNG ADULT;

ADOLESCENT; ADULT; AMINOPHENOLS; AMINOPYRIDINES; BASE SEQUENCE; BENZODIOXOLES; CHLORIDES; CYSTIC FIBROSIS; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; DOUBLE-BLIND METHOD; DRUG THERAPY, COMBINATION; FEMALE; FORCED EXPIRATORY VOLUME; HETEROZYGOTE; HOMOZYGOTE; HUMANS; MALE; QUINOLONES; SEQUENCE DELETION; SWEAT; YOUNG ADULT;

EID: 84904002908     PISSN: 22132600     EISSN: 22132619     Source Type: Journal    
DOI: 10.1016/S2213-2600(14)70132-8     Document Type: Article

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