LIONHEART, VISION-DMD and the Integration of Cardiac Outcomes in DMD Trials

This episode focuses on how the LIONHEART and VISION-DMD studies fit into the broader history of steroid use in Duchenne muscular dystrophy. Dr. Wittlieb-Weber reviews the well-established systemic and cardiac benefits of corticosteroids, including reductions in mortality and cardiomyopathy progression, while also addressing why newer agents aim to preserve benefit while mitigating long-term adverse effects.

She then discusses why cardiac outcomes and biomarkers are increasingly essential components of modern DMD trials. As survival improves, cardiac disease has emerged as a major driver of morbidity and mortality, often progressing silently over years. The conversation highlights the limitations of symptom-based cardiac endpoints and underscores the growing role of cardiac MRI, strain imaging, and biomarkers in capturing subtle but clinically meaningful changes that neurologists should be aware of when interpreting trial data.