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Urinary phenylacetylglutamine (U-PAGN) concentration as biomarker for adherence in patients with urea cycle disorders (UCD) treated with glycerol phenylbutyrate.
Mokhtarani, M; Diaz, G A; Lichter-Konecki, U; Berry, S A; Bartley, J; McCandless, S E; Smith, W; Harding, C; Le Mons, C; Coakley, D F; Lee, B; Scharschmidt, B F.
Affiliation
  • Mokhtarani M; Horizon Pharma, Brisbane, CA, USA.
  • Diaz GA; Mount Sinai School of Medicine, New York, NY, USA.
  • Lichter-Konecki U; Drexel University, Philadelphia, PA, USA.
  • Berry SA; University of Minnesota, Minneapolis, MN, USA.
  • Bartley J; Long Beach Memorial Hospital, Long Beach, CA, USA.
  • McCandless SE; Case Western Reserve University, Cleveland, OH, USA.
  • Smith W; Maine Medical Center, Portland, ME, USA.
  • Harding C; Oregon Health & Science University, Portland, OR, USA.
  • Le Mons C; National Urea Cycle Disorders Foundation, Pasadena, CA, USA.
  • Coakley DF; Horizon Pharma, Brisbane, CA, USA.
  • Lee B; Baylor College of Medicine, Houston, TX, USA.
  • Scharschmidt BF; Horizon Pharma, Brisbane, CA, USA.
Mol Genet Metab Rep ; 5: 12-14, 2015 Dec.
Article in En | MEDLINE | ID: mdl-28649536
ABSTRACT
Urinary phenylacetylglutamine (U-PAGN) concentrations in spot urine samples were analyzed as a dosing biomarker during glycerol phenylbutyrate (GPB) dosing in 68 healthy adults and 66 adult and pediatric patients with urea cycle disorders who participated in GPB clinical trials. Age- and body surface area (BSA)-specific 25th percentile cutoff points for spot U-PAGN concentrations (<~9000 µg/mL for < 2 years old patients, < 7000 µg/mL for > 2 years with BSA ≤ 1.3 m2, and <~5000 µg/mL for > 2 years of age with BSA > 1.3 m2) were determined as an approach to identify patients for whom increased dosing and/or adherence to prescribed dosing should be assessed.
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Full text: 1 Collection: 01-internacional Database: MEDLINE Language: En Journal: Mol Genet Metab Rep Year: 2015 Document type: Article Affiliation country: United States
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Full text: 1 Collection: 01-internacional Database: MEDLINE Language: En Journal: Mol Genet Metab Rep Year: 2015 Document type: Article Affiliation country: United States