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.
Preview |
Text
Muntoni_Safety and disease monitoring biomarkers in Duchenne muscular dystrophy- results from a Phase II trial_VoR.pdf - Published Version Download (819kB) | Preview |
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 |
Archive Staff Only
 |
View Item |
