Vamorolone

This sponsored series has explored how vamorolone is being integrated into modern Duchenne muscular dystrophy care, from clinical trial insights to real-world application. Through expert perspectives, the program has examined how evolving evidence is shaping steroid strategy within an increasingly complex and combination-driven treatment landscape.

As Duchenne muscular dystrophy management becomes more layered, long-term data remain essential to understanding how therapies perform beyond initial trials. In this final installment, Migvis Monduy, MD, pediatric neurologist and medical director of the Cerebral Palsy and Neuromotor Center at Nicklaus Children’s Hospital, reflects on the unanswered questions surrounding vamorolone and the broader steroid strategy in DMD care.

The conversation turns to durability, safety over years of exposure, and whether early biomarker signals translate into meaningful clinical outcomes such as reduced fractures, preserved growth trajectories, delayed cardiomyopathy, or improved survival. Monduy also considers how combination therapy, optimal timing of steroid initiation, and institutional variability may shape the next phase of evidence generation in Duchenne care.