DMD Treatment Algorithms: Initiating and Sequencing Therapies in New Patients

In this episode, the panel discusses how they approach treatment initiation and sequencing in newly diagnosed DMD patients. Dr. Veerapandiyan opens by acknowledging that the expanded therapeutic landscape, while encouraging, also makes initial clinic visits enormously complex. He separates the diagnostic disclosure visit from the treatment discussion, recognizing that families need time to process the diagnosis before absorbing information about multiple therapeutic options.

Corticosteroids remain his foundational therapy. From there, he considers the patient's age, genetic profile, and payer landscape. A key recurring question he raises with families is: "Should we act now, or wait for something better?" His consistent answer: since no one can predict when or whether a better option will emerge, he favors commercially approved therapies and available clinical trials over delay. He prioritizes microdystrophin gene therapy as an early goal, supplemented by exon skipping agents for eligible patients—though he tends to favor next-generation clinical trial options over first-generation weekly infusion agents due to the significant burden on families.

Dr. Brandsema adds important time-sensitive considerations: clinical trial eligibility may require specific treatment history, and patients can seroconvert to the viral vector used in gene therapy through community exposure, rendering them ineligible. These factors argue against indefinite delay in decision-making. At the same time, he cautions against catastrophizing urgency, noting that unlike spinal muscular atrophy, where time means motor neurons, in DMD time means muscle on a longer scale.

The panel also addresses the impact of socioeconomic and geographic barriers, with Dr. Veerapandiyan noting that for many Arkansas families, simply getting to clinic and affording medications represents the primary challenge. Dr. Chrzanowski references the observation that "your zip code is as important as your gene code." Finally, the discussion raises the complex question of re-dosing gene therapy, noting that it currently remains a one-and-done intervention, with promising but not yet practice-changing data on redosing.

In the next episode, "DMD Management: Evolving Regimens and Real-World Adherence," the panel explores how treatment approaches adapt over time and the practical challenges families face in maintaining complex therapeutic regimens.