Vamorolone’s Cardiac Rationale in Duchenne Muscular Dystrophy

In this episode, Carol Wittlieb-Weber, MD, provides cardiology context for why vamorolone’s pharmacologic profile is generating interest beyond skeletal muscle outcomes. She explains the role of aldosterone and mineralocorticoid receptor signaling in heart failure pathophysiology, emphasizing myocardial fibrosis as an early and critical feature of Duchenne cardiomyopathy. Drawing from landmark adult heart failure trials and Duchenne-specific cardiac studies, she outlines why MRA activity has become a foundational strategy in cardiac care.

The discussion then turns to how vamorolone’s dissociative corticosteroid properties may differ from traditional glucocorticoids with respect to blood pressure, fluid balance, and myocardial remodeling. Dr. Wittlieb-Weber highlights why the anti-fibrotic potential of MRA antagonism may be especially relevant when therapy is started early, and why serial cardiac MRI, rather than short-term hemodynamic changes, is likely to be the most meaningful way to assess long-term cardiac impact.