Vamorolone for treatment of Duchenne muscular dystrophy
Authors:
M. Rohlenová
Authors‘ workplace:
Klinika dětské neurologie 2. LF UK a FNMH – Motol, Praha
Published in:
Cesk Slov Neurol N 2026; 89(2): 87-92
Category:
Review Article
doi:
https://doi.org/10.48095/cccsnn202687
Overview
Corticosteroid therapy is currently the only therapeutic option for the treatment of Duchenne muscular dystrophy. However, long-term corticosteroid therapy is associated with numerous and serious side effects. At the end of 2023, the European Medicines Agency approved vamorolone, a partial agonist of the glucocorticoid receptor, which promises to maintain an anti-inflammatory effect while reducing the side effects of glucocorticoids. The article provides an overview of preclinical and clinical studies. The results show that vamorolone, especially at higher doses (6 mg/kg/day), has comparable efficacy to prednisone in maintaining muscle strength, has a demonstrably more favorable profile in maintaining growth rate, and potentially a lower incidence of undesirable psychological, bone, immunosuppressive, and cardiological effects, while body mass index values are comparable to prednisone, they do not increase over time.
Keywords:
Duchenne muscular dystrophy – vamorolone – corticotherapy
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Labels
Paediatric neurology Neurosurgery NeurologyArticle was published in
Czech and Slovak Neurology and Neurosurgery
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