Vamorolone, a synthetic steroid, is an orally available dissociative steroid with anti-inflammatory efficacy. It is a partial agonist of the glucocorticoid receptor (NR3C1) that appear to be safe and well tolerated. Also, vamorolone is a potent antagonist of the mineralocorticoid receptor (NR3C2). It is under development for children with Duchenne muscular dystrophy.
Cardiomyopathy is a leading cause of death for Duchenne muscular dystrophy. Here, we find that the mineralocorticoid receptor (MR) and glucocorticoid receptor (GR) can share common ligands but play distinct roles in dystrophic heart and skeletal muscle pathophysiology. Comparisons of
Proceedings of the National Academy of Sciences of the United States of America, 117(39), 24285-24293 (2020-09-13)
Duchenne muscular dystrophy is a genetic disorder that shows chronic and progressive damage to skeletal and cardiac muscle leading to premature death. Antiinflammatory corticosteroids targeting the glucocorticoid receptor (GR) are the current standard of care but drive adverse side effects
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