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

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. Although...

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Veröffentlicht in:Orphanet Journal of Rare Diseases 2021, Vol.16 (1)
Hauptverfasser: Kourakis, Stephanie, Timpani, Cara A, Campelj, Dean G, Hafner, Patricia, Gueven, Nuri, Fischer, Dirk, Rybalka, Emma
Format: Report
Sprache:eng
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Zusammenfassung: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. 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.
ISSN:1750-1172
1750-1172
DOI:10.1186/s13023-021-01758-9