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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...
Autores principales: | , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
BioMed Central
2021
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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. |
format | Online Article Text |
id | pubmed-7934375 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2021 |
publisher | BioMed Central |
record_format | MEDLINE/PubMed |
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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