Urinary phenylacetylglutamine (U-PAGN) concentration as biomarker for adherence in patients with urea cycle disorders (UCD) treated with glycerol phenylbutyrate

Autor: M. Mokhtarani, G.A. Diaz, U. Lichter-Konecki, S.A. Berry, J. Bartley, S.E. McCandless, W. Smith, C. Harding, C. Le Mons, D.F. Coakley, B. Lee, B.F. Scharschmidt
Jazyk: angličtina
Rok vydání: 2015
Předmět:
Zdroj: Molecular Genetics and Metabolism Reports, Vol 5, Iss C, Pp 12-14 (2015)
Druh dokumentu: article
ISSN: 2214-4269
DOI: 10.1016/j.ymgmr.2015.09.003
Popis: 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 (2 years with BSA ≤1.3 m2, and 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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