Evolving patient selection and clinical benefit criteria for sapropterin dihydrochloride (Kuvan®) treatment of PKU patients

Molecular Genetics and Metabolism

Volumn 105, Issue 4, 2012, Pages 672-676

  Gordon, Patricia ^a   Thomas, Janet A ^b   Suter, Ruth ^c   Jurecki, Elaina ^c  

^a PENN STATE CHILDREN S HOSPITAL   (United States)

^b UNIVERSITY OF COLORADO   (United States)

^c BIOMARIN PHARMACEUTICAL INC   (United States)

Author keywords

6R BH 4; 6R tetrahydrobiopterin; Kuvan; Phenylketonuria; PKU; Sapropterin

Indexed keywords

PHENYLALANINE; SAPROPTERIN; TYROSINE;

ADOLESCENT; ADULT; ARTICLE; BEHAVIOR; CHILD; DISEASE SEVERITY; DRUG CONTRAINDICATION; DRUG DOSE INCREASE; DRUG DOSE REDUCTION; DRUG DOSE TITRATION; DRUG INDICATION; FEMALE; FOLLOW UP; HUMAN; MALE; OUTCOME ASSESSMENT; PATIENT SELECTION; PHENYLKETONURIA; PREGNANCY; PRESCHOOL CHILD; PRIORITY JOURNAL; SCHOOL CHILD;

BIOPTERIN; CHILD, PRESCHOOL; DOSE-RESPONSE RELATIONSHIP, DRUG; FEMALE; HUMANS; INFANT; INFANT, NEWBORN; NITRIC OXIDE; PATIENT SELECTION; PHENYLALANINE; PHENYLKETONURIAS; PRACTICE GUIDELINES AS TOPIC; PROGNOSIS;

EID: 84863393087     PISSN: 10967192     EISSN: 10967206     Source Type: Journal    
DOI: 10.1016/j.ymgme.2011.12.023     Document Type: Article

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