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Safety and disease monitoring biomarkers in Duchenne muscular dystrophy: results from a Phase II trial

Wagner, KR; Guglieri, M; Ramaiah, SK; Charnas, L; Marraffino, S; Binks, M; Vaidya, VS; ... Muntoni, F; + view all (2021) Safety and disease monitoring biomarkers in Duchenne muscular dystrophy: results from a Phase II trial. Biomarkers in Medicine , 15 (15) pp. 1389-1396. 10.2217/bmm-2021-0222. Green open access

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Abstract

Aim: Evaluate the utility of glutamate dehydrogenase (GLDH) and cardiac troponin I as safety biomarkers, and creatine kinase and muscle injury panel as muscle health biomarkers in Duchenne muscular dystrophy. Patients & methods: Data were collected during a Phase II trial of domagrozumab. Results: GLDH was a more specific biomarker for liver injury than alanine aminotransferase. Cardiac troponin I elevations were variable and not sustained, limiting its applicability as a biomarker. Muscle injury panel biomarkers were no more informative than creatine kinase as a muscle health biomarker. Conclusion: Results support the use of GLDH as a specific biomarker for liver injury in patients with Duchenne muscular dystrophy.

Type: Article
Title: Safety and disease monitoring biomarkers in Duchenne muscular dystrophy: results from a Phase II trial
Open access status: An open access version is available from UCL Discovery
DOI: 10.2217/bmm-2021-0222
Publisher version: https://doi.org/10.2217/bmm-2021-0222
Language: English
Additional information: This work is licensed under the Creative Commons Attribution 4.0 License. To view a copy of this license, visit http://creativecommons.org/licenses/by/4.0/
Keywords: biomarker; cardiac troponin I; drug-induced liver injury; Duchenne muscular dystrophy; glutamate dehydrogenase
UCL classification: UCL
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health > Developmental Neurosciences Dept
URI: https://discovery-pp.ucl.ac.uk/id/eprint/10135825
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