Unmet Needs and Emerging Horizons for DMD

To start the discussion, Dr. Veerapandiyan highlighted several developments that stood out across the DMD therapeutic landscape at the annual 2026 MDA meeting. He noted encouraging long-term follow-up data for delandistrogene moxeparvovec, the approved gene therapy, where functional benefits are now becoming more apparent over time — a meaningful evolution given earlier concerns about the trial not meeting its primary endpoint. Real-world experience with givinostat across the three doses used in clinical practice, along with practical considerations for its use, also generated productive discussion. Despite the growing number of available options, Dr. Veerapandiyan emphasized a sobering reality: current therapies are largely stabilizing or slowing disease progression rather than producing measurable improvements, and that distinction remains an important goal for the field to work toward.

Drs. Chrzanowski and Brendsema raised the challenge of identifying appropriate biomarkers for DMD trials — whether functional measures, stride velocity, or objective imaging such as MRI — acknowledging that what works in a research setting is often impractical at the clinic level. Muscle MRIs and biopsies cannot realistically serve as routine efficacy monitors in clinical practice, highlighting the ongoing need to identify the most informative and accessible combination of outcome measures.

The panel paused to recognize the immense emotional and financial burden borne by families who participate in clinical trials, crediting them as the true drivers of progress in the field. Several underserved populations were identified as warranting greater attention, including non-ambulatory patients and those with Becker muscular dystrophy. The panel also looked ahead to the anticipated expansion of newborn screening for DMD, stressing the urgent need for Phase 4 studies and real-world registry data. Drawing on lessons from gene therapy toxicities identified only after broad clinical deployment in spinal muscular atrophy, the panel underscored that post-marketing surveillance is not optional — it is essential.

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