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Standard of care versus new-wave corticosteroids in the treatment of Duchenne muscular dystrophy: Can we do better?


Kourakis, S and Timpani, CA and Campelj, DG and Hafner, P and Gueven, N and Fischer, D and Rybalka, E, Standard of care versus new-wave corticosteroids in the treatment of Duchenne muscular dystrophy: Can we do better?, Orphanet Journal of Rare Diseases, 16, (1) pp. 1-9. ISSN 1750-1172 (2021) [Refereed Article]

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Copyright Statement

The Author(s) 2021. Open Access This article is licensed under a Creative CommonsAttribution 4.0 International (CC BY 4.0) License, ( which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made.

DOI: doi:10.1186/s13023-021-01758-9


Background:Pharmacological corticosteroid therapy is the standard of care in Duchenne Muscular Dystrophy (DMD) that aims to control symptoms and slow disease progression through potent anti-inflammatory action. However, a major concern is the significant adverse effects associated with long term-use.

MAIN: This review discusses the pros and cons of standard of care treatment for DMD and compares it to novel data generated with the new-wave dissociative corticosteroid, vamorolone. The current status of experimental anti-inflammatory pharmaceuticals is also reviewed, with insights regarding alternative drugs that could provide therapeutic advantage.

Conclusions: Although novel dissociative steroids may be superior substitutes to corticosteroids, other potential therapeutics should be explored. Repurposing or developing novel pharmacological therapies capable of addressing the many pathogenic features of DMD in addition to anti-inflammation could elicit greater therapeutic advantages.

Item Details

Item Type:Refereed Article
Keywords:Duchenne muscular dystrophy, corticosteroids, standard of care, anti-infammatory drugs, antiinfammation
Research Division:Biomedical and Clinical Sciences
Research Group:Pharmacology and pharmaceutical sciences
Research Field:Clinical pharmacology and therapeutics
Objective Division:Health
Objective Group:Clinical health
Objective Field:Treatment of human diseases and conditions
UTAS Author:Gueven, N (Dr Nuri Guven)
ID Code:147878
Year Published:2021
Web of Science® Times Cited:15
Deposited By:Pharmacy
Deposited On:2021-11-19
Last Modified:2021-12-08
Downloads:7 View Download Statistics

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