Cardiac Endpoints, Registries, and the Potential of Vamorolone in Becker Muscular Dystrophy

In the final episode, Dr. Wittlieb-Weber outlines what cardiologists are watching most closely as vamorolone moves into longer-term and post-approval studies. She discusses the importance of serial cardiac MRI, tracking fibrosis progression, ventricular function, and cardiac biomarkers such as NT-proBNP, while also emphasizing the value of large registries in understanding long-term outcomes and treatment interactions in real-world care.

The episode closes with a discussion of vamorolone’s evaluation in Becker muscular dystrophy and why cardiac risk should be viewed across the full dystrophinopathy spectrum. Dr. Wittlieb-Weber explains how patients with Becker may retain skeletal muscle function while developing severe cardiomyopathy, reinforcing the need for comprehensive cardiac assessment. For neurologists, the episode highlights how emerging data may help refine steroid decision-making and cardiac surveillance in both Duchenne and Becker populations.