Vamorolone: A Novel Steroid Option in Duchenne Muscular Dystrophy

Vamorolone represents a new class of steroid designed to provide the anti-inflammatory and muscle-protective benefits of traditional corticosteroids while minimizing common side effects such as growth suppression, weight gain, and behavioral changes. Compared with prednisone and deflazacort, vamorolone demonstrates a more favorable safety profile, particularly regarding bone health and metabolic effects. Its FDA approval was supported by clinical trial data showing improved motor function and reduced adverse events in boys with Duchenne muscular dystrophy. The LION-HEART study specifically evaluates vamorolone’s long-term safety, efficacy, and impact on cardiac function, addressing a critical aspect of DMD progression. Early results indicate maintained functional benefits alongside a reduction in steroid-related complications, supporting its potential as a safer long-term therapy. These findings provide clinicians with an important alternative for tailoring treatment to individual patient needs while mitigating the burdens associated with chronic steroid use.