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

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-us...

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Autores principales: Kourakis, Stephanie, Timpani, Cara A., Campelj, Dean G., Hafner, Patricia, Gueven, Nuri, Fischer, Dirk, Rybalka, Emma
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7934375/
https://www.ncbi.nlm.nih.gov/pubmed/33663533
http://dx.doi.org/10.1186/s13023-021-01758-9
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author Kourakis, Stephanie
Timpani, Cara A.
Campelj, Dean G.
Hafner, Patricia
Gueven, Nuri
Fischer, Dirk
Rybalka, Emma
author_facet Kourakis, Stephanie
Timpani, Cara A.
Campelj, Dean G.
Hafner, Patricia
Gueven, Nuri
Fischer, Dirk
Rybalka, Emma
author_sort Kourakis, Stephanie
collection PubMed
description 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.
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spelling pubmed-79343752021-03-08 Standard of care versus new-wave corticosteroids in the treatment of Duchenne muscular dystrophy: Can we do better? Kourakis, Stephanie Timpani, Cara A. Campelj, Dean G. Hafner, Patricia Gueven, Nuri Fischer, Dirk Rybalka, Emma Orphanet J Rare Dis Position Statement 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. BioMed Central 2021-03-04 /pmc/articles/PMC7934375/ /pubmed/33663533 http://dx.doi.org/10.1186/s13023-021-01758-9 Text en © The Author(s) 2021 Open AccessThis article is licensed under a Creative Commons Attribution 4.0 International 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. The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article's Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/. The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated in a credit line to the data.
spellingShingle Position Statement
Kourakis, Stephanie
Timpani, Cara A.
Campelj, Dean G.
Hafner, Patricia
Gueven, Nuri
Fischer, Dirk
Rybalka, Emma
Standard of care versus new-wave corticosteroids in the treatment of Duchenne muscular dystrophy: Can we do better?
title Standard of care versus new-wave corticosteroids in the treatment of Duchenne muscular dystrophy: Can we do better?
title_full Standard of care versus new-wave corticosteroids in the treatment of Duchenne muscular dystrophy: Can we do better?
title_fullStr Standard of care versus new-wave corticosteroids in the treatment of Duchenne muscular dystrophy: Can we do better?
title_full_unstemmed Standard of care versus new-wave corticosteroids in the treatment of Duchenne muscular dystrophy: Can we do better?
title_short Standard of care versus new-wave corticosteroids in the treatment of Duchenne muscular dystrophy: Can we do better?
title_sort standard of care versus new-wave corticosteroids in the treatment of duchenne muscular dystrophy: can we do better?
topic Position Statement
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7934375/
https://www.ncbi.nlm.nih.gov/pubmed/33663533
http://dx.doi.org/10.1186/s13023-021-01758-9
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