Episode 160: Early Pathology, Biomarkers, and the Next Phase of DMD Care

Episode 160 of the NeurologyLive^® Mind Moments^® podcast is now live! Scroll down to listen or click here to subscribe on your favorite streaming service.

The Mind Moments podcast features exclusive interviews with leaders in the field discussing the latest research and disease management strategies across the breadth of neurology, including epilepsy, multiple sclerosis, Parkinson disease, dementia, sleep disorders, and more.

In this Mind Moments episode, Jeff Chamberlain, PhD, joins the podcast during Duchenne Muscular Dystrophy Awareness Week to provide clinical and translational perspective on the evolving landscape of DMD biology and therapy. Chamberlain, professor at the University of Washington School of Medicine and Director of the Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center in Seattle, reflects on aspects of Duchenne pathophysiology that may still be underappreciated, including evidence that disease processes begin earlier than once recognized and the growing importance of immunologic factors in shaping progression and therapeutic response. The conversation also explores how neuromuscular specialists should approach treatment timing and combination strategies as gene-targeted therapies expand, the evolving interpretation and limitations of biomarkers such as creatine kinase and dystrophin expression, and what emerging gene therapy platforms may signal for care heading into 2026 and beyond.

The stories featured in this week’s Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here:

• Regenxbio's MPS II Gene Therapy RGX-121 Hit With CRL
• FDA Accepts New Drug Application for Orexin Agonist Oveporexton in Narcolepsy Type 1, Grants Priority Review
• FDA Expands Indication for Pitolisant to Treat Cataplexy in Pediatric Narcolepsy

The 2026 MDA Conference kicks off March 8 in Orlando, Florida! For more information about the event, click here.

EPISODE BREAKDOWN

• 1:15 – Underrecognized aspects of DMD pathophysiology, including early onset and immunologic drivers
• 4:50 – Treatment timing, sequencing, and the rationale for combination strategies
• 8:00 – Neurology News Minute
• 10:30 – Clinical trial and real-world implications of dystrophin and CK as biomarkers
• 16:20 – Anticipated gene therapy innovation and safety considerations heading into 2026

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