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Discovery of potential urine-accessible metabolite biomarkers associated with muscle disease and corticosteroid response in the mdx mouse model for Duchenne

Mathula Thangarajh, Aiping Zhang, Kirandeep Gill, Habtom W Ressom, Zhenzhi Li, Rency S Varghese, Eric P Hoffman, Kanneboyina Nagaraju, Yetrib Hathout and Simina M Boca

PLOS ONE, 2019, vol. 14, issue 7, 1-17

Abstract: Urine is increasingly being considered as a source of biomarker development in Duchenne Muscular Dystrophy (DMD), a severe, life-limiting disorder that affects approximately 1 in 4500 boys. In this study, we considered the mdx mice—a murine model of DMD—to discover biomarkers of disease, as well as pharmacodynamic biomarkers responsive to prednisolone, a corticosteroid commonly used to treat DMD. Longitudinal urine samples were analyzed from male age-matched mdx and wild-type mice randomized to prednisolone or vehicle control via liquid chromatography tandem mass spectrometry. A large number of metabolites (869 out of 6,334) were found to be significantly different between mdx and wild-type mice at baseline (Bonferroni-adjusted p-value

Date: 2019
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Persistent link: https://EconPapers.repec.org/RePEc:plo:pone00:0219507

DOI: 10.1371/journal.pone.0219507

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