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

• Main Authors: Stephanie Kourakis, Cara A. Timpani, Dean G. Campelj, Patricia Hafner, Nuri Gueven, Dirk Fischer, Emma Rybalka
• Format: Article
• Language: English
• Published: BMC 2021-03-01
• Series: Orphanet Journal of Rare Diseases
• Subjects: Duchenne muscular dystrophy; Corticosteroids; Standard of care; Anti-inflammatory drugs; Anti-inflammation
• Online Access: https://doi.org/10.1186/s13023-021-01758-9

Abstract 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.