Angela Lek, PhD, on the Scientific Priorities that Shaped the 2026 MDA Conference

“We're always covering the cutting edge topic—what’s important to have conversations around—not just the readouts for clinical trials, but the really exciting technologies that are emerging around the corner.”

The Muscular Dystrophy Association (MDA), an organization dedicated to serving the community of patients with neuromuscular disorders, including muscular dystrophies and other types of neuromuscular disease, holds its Clinical & Scientific Conference each year to bring together physicians, clinical trial investigators, academic researchers, patient advocates, biotech executives, and other stakeholders to share and discuss the latest findings in neuromuscular disease research. The conference this year was held from March 8 to 11, 2026, in Orlando, Florida.

On Tuesday, March 10, CGTLive® sat down with Angela Lek, PhD, the chief research officer of MDA, to get her insight on highlights and themes from the conference thus far. Lek highlighted several cross-cutting priorities, beginning with muscle regeneration and repair, a topic she described as relevant to many of the more than 300 neuromuscular conditions MDA covers, with sessions exploring cell therapy strategies to restore muscle lost to disease or injury. Disease-specific spotlights this year included limb-girdle muscular dystrophy, titanopathies, infantile-onset muscular dystrophies, and autoimmune neuromuscular conditions. She noted that sessions on amyotrophic lateral sclerosis genetics and translational research were also featured.

Among the moments Lek expressed particular enthusiasm for was a session on nonviral delivery vehicles for genetic medicines, noting that meaningful progress has occurred in the field since MDA convened on the topic several years ago. She emphasized that nonviral approaches could enable redosing and potentially reduce immunogenicity—practical advantages over current viral vector platforms. A complementary session on immune responses to approved and investigational gene therapies, including onasemnogene abeparvovec (Zolgensma) and delandistrogene moxeparvovec (Elevidys), was also featured, with presentations covering mechanistic studies and mitigation strategies.

Lek also touched on drug development access and economics, noting that of the 300-plus conditions MDA covers, the majority are ultra-rare (defined as fewer than 1000 affected patients) with only approximately 10 considered commercially viable. A related session examined regulatory and investor landscape factors influencing therapeutic development beyond the science itself.

Click here for more MDA 2026 coverage.