Ataluren (PTC124) induces cystic fibrosis transmembrane conductance regulator protein expression and activity in children with nonsense mutation cystic fibrosis

American Journal of Respiratory and Critical Care Medicine

Volumn 182, Issue 10, 2010, Pages 1262-1272

  Sermet Gaudelus, Isabelle ^a   De Boeck, Kris ^b   Casimir, Georges J ^c   Vermeulen, François ^b   Leal, Teresinha ^d   Mogenet, Agnès ^a   Roussel, Delphine ^a   Fritsch, Janine ^a   Hanssens, Laurence ^c   Hirawat, Samit ^e   Miller, Nilsen L ^e   Constantine, Scott ^e   Reha, Allen ^e   Ajayi, Temitayo ^e   Elfring, Gary L ^e   Miller, Langdon L ^e  

^a HÔPITAL NECKER ENFANTS MALADES   (France)

^b UNIVERSITY OF LEUVEN   (Belgium)

^c HOP UNIV DES ENFANTS REINE FABIOLA   (Belgium)

^d UNIVERSITÉ CATHOLIQUE DE LOUVAIN   (Belgium)

^e PTC THERAPEUTICS   (United States)

Author keywords

Codon, nonsense; Cystic fibrosis transmembrane conductance regulator; Drugs, investigational; Membrane potentials; Pediatrics

Indexed keywords

ATALUREN; TRANSMEMBRANE CONDUCTANCE REGULATOR;

ABDOMINAL PAIN; ABNORMAL LABORATORY RESULT; ADOLESCENT; ADULT; ALANINE AMINOTRANSFERASE BLOOD LEVEL; ALBUMIN BLOOD LEVEL; ARTHRALGIA; ARTICLE; ASCITES; ASPARTATE AMINOTRANSFERASE BLOOD LEVEL; ASTHENIA; BACTERIAL INFECTION; BICARBONATE BLOOD LEVEL; BLOODY DIARRHEA; BODY WEIGHT; CALCIUM BLOOD LEVEL; CHILD; CHLORIDE BLOOD LEVEL; CLINICAL ARTICLE; CLINICAL TRIAL; CONSTIPATION; CONTROLLED CLINICAL TRIAL; CONTROLLED STUDY; CORTICOTROPIN BLOOD LEVEL; COUGHING; CREATINE KINASE BLOOD LEVEL; CROSSOVER PROCEDURE; CYSTIC FIBROSIS; DIARRHEA; DOSE RESPONSE; DRUG ACTIVITY; DRUG BLOOD LEVEL; DRUG DOSE REDUCTION; DRUG SAFETY; DRUG TOLERABILITY; DYSPHAGIA; ESOPHAGUS VARICES; FATIGUE; FEMALE; FEVER; GLUCOSE BLOOD LEVEL; HEADACHE; HEMATEMESIS; HEMOGLOBIN BLOOD LEVEL; HUMAN; HYDROCORTISONE BLOOD LEVEL; LACTATE DEHYDROGENASE BLOOD LEVEL; LARYNGITIS; LEUKOCYTE COUNT; LIPOPROTEIN BLOOD LEVEL; MALE; MULTICENTER STUDY; MULTIPLE CYCLE TREATMENT; NEUTROPHIL COUNT; NONSENSE MUTATION; NOSE CONGESTION; PALATABILITY; PATIENT COMPLIANCE; PHARMACODYNAMICS; POTASSIUM BLOOD LEVEL; PRESCHOOL CHILD; PRIORITY JOURNAL; PROTEIN EXPRESSION; PRURITUS; RANDOMIZED CONTROLLED TRIAL; RASH; RHINITIS; RHINOPHARYNGITIS; SCHOOL CHILD; SIDE EFFECT; SODIUM BLOOD LEVEL; TENDINITIS; THIRST; THORAX PAIN; TRIACYLGLYCEROL BLOOD LEVEL; UREA NITROGEN BLOOD LEVEL; URIC ACID BLOOD LEVEL; VOMITING;

ADOLESCENT; CHILD; CODON, NONSENSE; CROSS-OVER STUDIES; CYSTIC FIBROSIS; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; DOSE-RESPONSE RELATIONSHIP, DRUG; FEMALE; GENE EXPRESSION REGULATION; HUMANS; MALE; NASAL MUCOSA; OXADIAZOLES;

EID: 78349290383     PISSN: 1073449X     EISSN: 15354970     Source Type: Journal    
DOI: 10.1164/rccm.201001-0137OC     Document Type: Article

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