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


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Kourakis, S, Timpani, CA, Campelj, DG, Hafner, P, Gueven, N ORCID: 0000-0003-3782-767X, Fischer, D and Rybalka, E 2021 , 'Standard of care versus new-wave corticosteroids in the treatment of Duchenne muscular dystrophy: Can we do better?' , Orphanet Journal of Rare Diseases, vol. 16, no. 1 , pp. 1-9 , doi: 10.1186/s13023-021-01758-9.

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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 Type: Article
Authors/Creators:Kourakis, S and Timpani, CA and Campelj, DG and Hafner, P and Gueven, N and Fischer, D and Rybalka, E
Keywords: Duchenne muscular dystrophy, corticosteroids, standard of care, anti-infammatory drugs, antiinfammation
Journal or Publication Title: Orphanet Journal of Rare Diseases
Publisher: BioMed Central
ISSN: 1750-1172
DOI / ID Number: 10.1186/s13023-021-01758-9
Copyright Information:

© 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.

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