Title

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

Document Type

Article

Publication Date

9-2015

Institution/Department

MMCRI

Journal Title

Molecular genetics and metabolism reports

ISSN

2214-4269

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

First Page

12

Last Page

14

Share

COinS