Long-term developmental progression in infants and young children taking sapropterin for phenylketonuria: A two-year analysis of safety and efficacy

Genetics in Medicine

Volumn 17, Issue 5, 2015, Pages 365-373

  Longo, Nicola ^a   Siriwardena, Komudi ^b   Feigenbaum, Annette ^b   Dimmock, David ^c   Burton, Barbara K ^d   Stockler, Sylvia ^e   Waisbren, Susan ^f   Lang, William ^g   Jurecki, Elaina ^g   Zhang, Charlie ^g   Prasad, Suyash ^g  

^a UNIVERSITY OF UTAH SCHOOL OF MEDICINE   (United States)

^b HOSPITAL FOR SICK CHILDREN   (Canada)

^c MEDICAL COLLEGE OF WISCONSIN   (United States)

^d CHILDREN'S MEMORIAL HOSPITAL   (United States)

^e BC CHILDREN S HOSPITAL   (Canada)

^f BOSTON CHILDREN S HOSPITAL   (United States)

^g BIOMARIN PHARMACEUTICAL INC   (United States)

Author keywords

Children; pediatric; phenylketonuria; sapropterin; treatment

Indexed keywords

PHENYLALANINE; SAPROPTERIN; BIOPTERIN;

ABDOMINAL PAIN; ARTICLE; BAYLEY SCALES OF INFANT DEVELOPMENT; CHILD; CHILD DEVELOPMENT; COGNITION ASSESSMENT; CONSTIPATION; CROUP; DIARRHEA; DIETARY INTAKE; DRUG DOSE REDUCTION; DRUG EFFICACY; DRUG INDUCED HEADACHE; DRUG SAFETY; DRUG TOLERABILITY; EAR INFECTION; FEMALE; GROUPS BY AGE; HUMAN; INFANT; INJURY; INTELLIGENCE QUOTIENT; LOCAL THERAPY; LONG TERM CARE; MAJOR CLINICAL STUDY; MALE; NEWBORN; NOSE OBSTRUCTION; OPEN STUDY; PHENYLKETONURIA; PNEUMONIA; PRESCHOOL CHILD; SEIZURE; TREATMENT RESPONSE; UPPER ABDOMINAL PAIN; UPPER RESPIRATORY TRACT INFECTION; VOMITING; ANALOGS AND DERIVATIVES; BLOOD; CLINICAL TRIAL; COGNITION; DISEASE COURSE; DRUG EFFECTS; MULTICENTER STUDY; PHENYLKETONURIAS; PROSPECTIVE STUDY; TIME FACTOR; TREATMENT OUTCOME;

BIOPTERIN; CHILD; CHILD DEVELOPMENT; CHILD, PRESCHOOL; COGNITION; DISEASE PROGRESSION; FEMALE; HUMANS; INFANT; INFANT, NEWBORN; MALE; PHENYLALANINE; PHENYLKETONURIAS; PROSPECTIVE STUDIES; TIME FACTORS; TREATMENT OUTCOME;

EID: 84928880172     PISSN: 10983600     EISSN: 15300366     Source Type: Journal    
DOI: 10.1038/gim.2014.109     Document Type: Article

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