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

Authors

M Mokhtarani
G A Diaz
U Lichter-Konecki
S A Berry
J Bartley
S E McCandless
W Smith, Maine Medical Center
C Harding
C Le Mons
D F Coakley
B Lee
B F Scharschmidt

Document Type

Article

Publication Date

9-2015

Institution/Department

MMCRI

Journal Title

Molecular genetics and metabolism reports

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 (2 years with BSA ≤ 1.3 m

ISSN

2214-4269

First Page

12

Last Page

14

Recommended Citation

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; and Scharschmidt, B F, "Urinary phenylacetylglutamine (U-PAGN) concentration as biomarker for adherence in patients with urea cycle disorders (UCD) treated with glycerol phenylbutyrate." (2015). MaineHealth Maine Medical Center. 396.
https://knowledgeconnection.mainehealth.org/mmc/396