Ataluren for the treatment of nonsense-mutation cystic fibrosis: A randomised, double-blind, placebo-controlled phase 3 trial

The Lancet Respiratory Medicine

Volumn 2, Issue 7, 2014, Pages 539-547

  Kerem, Eitan ^a   Konstan, Michael W ^b   De Boeck, Kris ^c   Accurso, Frank J ^d   Sermet Gaudelus, Isabelle ^e   Wilschanski, Michael ^a   Elborn, J Stuart ^f   Melotti, Paola ^g   Bronsveld, Inez ^h   Fajac, Isabelle ^i,j   Malfroot, Anne ^k   Rosenbluth, Daniel B ^l   Walker, Patricia A ^m   McColley, Susanna A ⁿ   Knoop, Christiane ^o   Quattrucci, Serena ^p   Rietschel, Ernst ^q   Zeitlin, Pamela L ^r   Barth, Jay ^s   Elfring, Gary L ^s   Welch, Ellen M ^s   Branstrom, Arthur ^s   Spiegel, Robert J ^s   Peltz, Stuart W ^s   Ajayi, Temitayo ^s   Rowe, Steven M ^t   more..

^a HADASSAH HEBREW UNIVERSITY MEDICAL CENTER   (Israel)

^b CASE WESTERN RESERVE UNIVERSITY   (United States)

^c UNIVERSITY OF LEUVEN   (Belgium)

^d CHILDREN S HOSPITAL COLORADO   (United States)

^e HÔPITAL NECKER ENFANTS MALADES   (France)

^f QUEEN'S UNIVERSITY BELFAST   (United Kingdom)

^g UNIVERSITY HOSPITAL OF VERONA   (Italy)

^h UNIVERSITY MEDICAL CENTER UTRECHT   (Netherlands)

ⁱ UNIVERSITÉ PARIS DESCARTES   (France)

^j université Paris Cité   (France)

^k UNIVERSITY HOSPITAL BRUSSELS   (Belgium)

^l WASHINGTON UNIVERSITY SCHOOL OF MEDICINE   (United States)

^m MOUNT SINAI HOSPITAL   (United States)

ⁿ NORTHWESTERN UNIVERSITY   (United States)

^o ERASME HOSPITAL   (Belgium)

^p SAPIENZA UNIVERSITY OF ROME   (Italy)

^q UNIVERSITY HOSPITAL OF COLOGNE   (Germany)

^r JOHNS HOPKINS UNIVERSITY   (United States)

^s PTC THERAPEUTICS   (United States)

^t UNIVERSITY OF ALABAMA SCHOOL OF MEDICINE   (United States)

more..

Author keywords

[No Author keywords available]

Indexed keywords

ATALUREN; CREATININE; PLACEBO; TOBRAMYCIN; ANTIINFECTIVE AGENT; CFTR PROTEIN, HUMAN; CHLORIDE; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; OXADIAZOLE DERIVATIVE; STOP CODON;

ABDOMINAL PAIN; ACUTE KIDNEY FAILURE; ADOLESCENT; ADULT; ARTICLE; CHILD; CONGENITAL MALFORMATION; CONTROLLED STUDY; CYSTIC FIBROSIS; DIABETES MELLITUS; DIARRHEA; DISEASE EXACERBATION; DOUBLE BLIND PROCEDURE; DRUG EFFICACY; DRUG FATALITY; DRUG SAFETY; DRUG WITHDRAWAL; EUROPE; EVENING DOSAGE; FEMALE; FEVER; FORCED EXPIRATORY VOLUME; GENETIC DISORDER; HEADACHE; HEMOPTYSIS; HUMAN; HYPERCREATININEMIA; KIDNEY DISEASE; MAJOR CLINICAL STUDY; MALE; MORNING DOSAGE; MULTICENTER STUDY; NEPHROLITHIASIS; NONSENSE MUTATION; NONSENSE MUTATION CYSTIC FIBROSIS; NORTH AMERICA; OUTCOME ASSESSMENT; PANCREATITIS; PHASE 3 CLINICAL TRIAL; PRIORITY JOURNAL; RANDOMIZED CONTROLLED TRIAL; RASH; SKIN DISEASE; SPIROMETRY; URINARY TRACT INFECTION; CHEMICALLY INDUCED; CHEMISTRY; CLINICAL TRIAL; DISEASE COURSE; GENETICS; MIDDLE AGED; PATHOPHYSIOLOGY; STOP CODON; SWEAT; YOUNG ADULT;

ACUTE KIDNEY INJURY; ADOLESCENT; ADULT; ANTI-BACTERIAL AGENTS; CHILD; CHLORIDES; CODON, NONSENSE; CYSTIC FIBROSIS; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; DISEASE PROGRESSION; DOUBLE-BLIND METHOD; FEMALE; FORCED EXPIRATORY VOLUME; HUMANS; MALE; MIDDLE AGED; OXADIAZOLES; SWEAT; TOBRAMYCIN; YOUNG ADULT;

EID: 84904016376     PISSN: 22132600     EISSN: 22132619     Source Type: Journal    
DOI: 10.1016/S2213-2600(14)70100-6     Document Type: Article

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