Journal article

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

Stephanie Kourakis, Cara A Timpani, Dean G Campelj, Patricia Hafner, Nuri Gueven, Dirk Fischer, Emma Rybalka

ORPHANET JOURNAL OF RARE DISEASES | BMC | Published : 2021

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 st..

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University of Melbourne Researchers