On March 8-11, 2026, the Muscular Dystrophy Association hosted their annual Clinical & Scientific Conference in Orlando, Florida. The event, which hosted over 2,000 attendees, brought scientific experts and patients together to discuss recent advancements in research and care for individuals with neuromuscular diseases, including Duchenne muscular dystrophy (DMD).
Delandistrogene moxeparvovec has manageable safety profile
An analysis of several clinical trials of delandistrogene moxeparvovec, marketed as Elevidys, found that the safety profile of the gene therapy is generally manageable, though care must be taken to observe patients for serious adverse events.
The most common side effects included vomiting (58.5%), nausea (36.0%) and poor appetite (28.8%). In addition, two treatment-related deaths from acute liver failure have occurred within 90 days of dosing.
Read more about DMD therapies
The researchers emphasize the importance of carefully monitoring patient health, particularly within the first three months after infusion.
New microdystrophin gene therapy holds promise for DMD
SGT-003, a microdystrophin gene therapy, improved muscle health and heart function in a phase 1/2 clinical trial of 39 ambulatory children with DMD.
Biopsies from participants at Day 90 confirmed expression of microdystrophin in the muscle tissue, with evidence of restored muscle cell membranes. Ejection fractions, a measure of how well the left ventricle pumps blood, also increased over a 12-month period.
The study’s investigators hope to expand the eligibility criteria to include older and nonambulatory patients.
Givinostat safe for long-term use in patients with DMD
A preliminary analysis of an ongoing open-label extension revealed no new safety concerns associated with givinostat, an oral drug that reduces muscle inflammation.
Of the 207 patients included, 98.1% reported at least one treatment-emergent adverse event. The majority were mild to moderate. Some participants experienced initial decreases in platelets and increases in triglycerides, though levels of both stabilized after a few weeks of treatment.
Corticosteroids remain key to DMD care
Corticosteroid treatment for DMD has developed significantly over time, Dr. Ezgi Saylam explained during a session at the MDA Conference. Even with the advent of novel therapies, corticosteroids form the foundation of care for many patients.
Prednisone and deflazacort are commonly prescribed to individuals with DMD, though both carry risks of long-term side effects. Vamorolone, a newer steroid that was approved in October 2023, was designed to carry fewer side effects than existing options. More research is needed to understand its efficacy, though.
Researchers are also experimenting with dosing regimens, Saylam stated. While daily treatment is common, prescribing corticosteroids for weekend use only may reduce the risk of long-term damage.
Exon-skipping therapy offers cardiopulmonary and muscular benefits
Zeleciment rostudirsen (also known as DYNE-251 or z-rostudirsen), an exon 51-skipping therapy, improved muscle, heart and lung function in the DELIVER trial of patients with DMD.
After 24 months, participants reported improvements in time to rise, 10 meter walk test and percent predicted forced vital capacity. Cardiac magnetic resonance imaging also suggested that DYNE-251 may preserve contractile quality.
Data as of August 19, 2025, supported the safety and tolerability of DYNE-251.
Sign up here to get the latest news, perspectives, and information about DMD sent directly to your inbox. Registration is free and only takes a minute.
