Vamorolone in the Era of Gene Therapy

As therapeutic options for Duchenne muscular dystrophy continue to expand, this sponsored series explores how vamorolone fits within an increasingly complex treatment landscape. Through conversations with specialists across disciplines, the program examines how clinical data and real-world experience are shaping the role of corticosteroids alongside emerging disease-modifying approaches.

The arrival of gene therapy has reshaped expectations in Duchenne muscular dystrophy, but it has not eliminated the need for corticosteroids. In this episode, Migvis Monduy, MD, pediatric neurologist and medical director of the Cerebral Palsy and Neuromotor Center at Nicklaus Children’s Hospital, discusses how vamorolone remains relevant even as mutation-specific and gene-based therapies advance.

Monduy reflects on the practical realities of steroid continuation during and after gene therapy infusion, the need for higher periprocedural dosing, and the absence of formal clinical trial data evaluating vamorolone as baseline therapy in pivotal gene therapy studies. The conversation offers perspective on how clinicians are approaching these layered treatment strategies in real-world settings while longer-term evidence continues to emerge.