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Urinary phenylacetylglutamine (U-PAGN) concentration as biomarker for adherence in patients with urea cycle disorders (UCD) treated with glycerol phenylbutyrate
Journal article   Open access   Peer reviewed

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

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, …
Molecular genetics and metabolism reports, v 5(C)
Dec 2015
DOI: https://doi.org/10.1016/j.ymgmr.2015.09.003
PMID: 28649536
Featured in Collection :   UN Sustainable Development Goals @ Drexel
url
https://doi.org/10.1016/j.ymgmr.2015.09.003View
Published, Version of Record (VoR)CC BY-NC-ND V4.0,  Open

Abstract

Biomarker Glycerol phenylbutyrate Phenylacetate Phenylbutyrate Urea cycle disorder Urinary phenylacetylglutamine
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 (<~9000μg/mL for <2years old patients, <7000μg/mL for >2years with BSA ≤1.3m2, and <~5000μg/mL for >2years of age with BSA >1.3m2) 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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5 citations in Web of Science
6 citations in Scopus

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#3 Good Health and Well-Being

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Domestic collaboration
Web of Science research areas
Genetics & Heredity
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