The response of patients with phenylketonuria and elevated serum phenylalanine to treatment with oral sapropterin dihydrochloride (6R-tetrahydrobiopterin): A phase II, multicentre, open-label, screening study

Journal of Inherited Metabolic Disease

Volumn 30, Issue 5, 2007, Pages 700-707

  Burton, B K ^a,m   Grange, D K ^b   Milanowski, A ^c   Vockley, G ^d   Feillet, F ^e   Crombez, E A ^f   Abadie, V ^g   Harding, C O ^h   Cederbaum, S ⁱ   Dobbelaere, D ^j   Smith, A ^k   Dorenbaum, A ^l  

^a NORTHWESTERN UNIVERSITY   (United States)

^b ST LOUIS CHILDREN S HOSPITAL   (United States)

^c INSTITUTE OF MOTHER AND CHILD   (Poland)

^d CHILDREN S HOSPITAL OF PITTSBURGH   (United States)

^e HÔPITAL D ENFANTS   (France)

^f UNIVERSITY OF CALIFORNIA   (United States)

^g HÔPITAL NECKER ENFANTS MALADES   (France)

^h OREGON HEALTH AND SCIENCE UNIVERSITY   (United States)

ⁱ UCLA MEDICAL CENTER   (United States)

^j LILLE UNIVERSITY HOSPITAL   (France)

^k Pacific Data Designs   (United States)

^l BIOMARIN PHARMACEUTICAL INC   (United States)

^m CHILDREN'S MEMORIAL HOSPITAL   (United States)

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Author keywords

[No Author keywords available]

Indexed keywords

PHENYLALANINE; SAPROPTERIN;

ABDOMINAL PAIN; ADOLESCENT; ADULT; AMINO ACID BLOOD LEVEL; ARTICLE; CLINICAL TRIAL; COHORT ANALYSIS; DECREASED APPETITE; DIARRHEA; DIET RESTRICTION; DRUG RESPONSE; DRUG SAFETY; DRUG TOLERABILITY; FATIGUE; FEMALE; FLATULENCE; HEADACHE; HUMAN; HYPERREFLEXIA; LARYNX PAIN; MAJOR CLINICAL STUDY; MALE; MIGRAINE; MULTICENTER STUDY; NAUSEA; OPEN STUDY; OUTCOME ASSESSMENT; PATIENT COMPLIANCE; PATIENT IDENTIFICATION; PATIENT SELECTION; PHASE 2 CLINICAL TRIAL; PHENYLKETONURIA; SCHOOL CHILD; SORE THROAT; THROMBOCYTOPENIA; TREATMENT DURATION; TREMOR; UPPER RESPIRATORY TRACT INFECTION; VOMITING;

ADMINISTRATION, ORAL; ADOLESCENT; ADULT; BIOPTERIN; CHILD; EUROPE; FEMALE; HUMANS; MALE; MIDDLE AGED; NORTH AMERICA; PHENYLALANINE; PHENYLKETONURIAS; TIME FACTORS; TREATMENT OUTCOME; UP-REGULATION;

EID: 35248882919     PISSN: 01418955     EISSN: 15732665     Source Type: Journal    
DOI: 10.1007/s10545-007-0605-z     Document Type: Article

References (20)

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