Reframing Steroid Therapy in Neuromuscular Disease: Vamorolone and LIONHEART

In this epiosde, Carol Wittlieb-Weber, MD, provides a cardiology-focused look at why vamorolone’s dissociative steroid profile and mineralocorticoid receptor antagonism may matter for cardiac fibrosis and remodeling in DMD.

In this segment, Wittlieb-Leber examines how vamorolone trials address long-standing questions about steroid exposure, cardiac risk, and meaningful cardiac endpoints in Duchenne.

A forward-looking discussion on cardiac monitoring strategies, real-world data, and how vamorolone may apply across Duchenne and Becker muscular dystrophy.

Migvis Monduy, MD, provided clinical perspective on how vamorolone compares with traditional corticosteroids and why its safety profile matters in long-term Duchenne management.

A closer look at how LIONHEART and VISION-DMD reinforce vamorolone’s distinct profile in Duchenne muscular dystrophy

A practical discussion on how vamorolone is being incorporated into Duchenne management, including its role in steroid initiation, reintroduction after prior intolerance, and long-term planning across different stages of disease.

A forward-looking discussion on the continued role of vamorolone as background steroid therapy in patients receiving gene or mutation-specific treatments, including what is known, what remains uncertain, and how clinicians are navigating combination strategies in practice.

In this final episode, Migvis Monduy, MD, discussed long-term data needed to define vamorolone’s durability, safety, and impact across disease stages, including unanswered questions around bone health, growth, cardiomyopathy, and combination strategies.