DMD Management: Evolving Regimens and Real-World Adherence

This episode examines how treatment regimens are adapted over the disease course and how real-world factors shape clinical decision-making. Dr. Jayaraman notes that for older patients, weekend corticosteroid dosing may be more tolerable and sustainable than daily dosing—and that any steroid is better than none. She reiterates population-level data supporting steroid use: approximately three additional years of ambulation, improved cardiac and pulmonary function, and reduced scoliosis surgery rates.

The discussion highlights the growing complexity of treatment monitoring. Therapies such as givinostat and gene therapy require frequent clinic visits and laboratory monitoring. For families who can barely make it to clinic once a year, these burdens can make otherwise promising therapies impractical. Dr. Chrzanowski describes tailoring therapy choices to what a given family can realistically sustain—a principle of "do no harm" applied to access and compliance.

A patient case is discussed in which gradual escalation of corticosteroid dosing—from weekend prednisone to daily deflazacort—appears to coincide with stabilization of a patient who had been showing functional decline. The panel uses this to illustrate the real-time learning process in DMD care.

The episode then pivots to gene therapy access, revisiting the brief period during which delandistrogene moxeparvovec was available for non-ambulatory patients under accelerated approval. Dr. Veerapandiyan describes his experience counseling families about the limited efficacy data and emerging safety signals in this population. The discussion also touches on the importance of shared decision-making with older patients who can assert their own preferences, contrasting this with the parent-driven decisions made for young children. The panel notes that while gene therapy is currently one-and-done, re-dosing research is actively ongoing.

In the next episode, "Emerging Therapies in DMD," the panel highlights the most promising investigational therapies and what the field can anticipate in the years ahead.