Scientific Priorities that Shaped MDA's 2026 Meeting: Angela Lek, PhD

This interview was originally published on our sister site, CGTLive®.

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“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), which serves patients living with neuromuscular disorders—among them muscular dystrophies and a broad range of related neuromuscular diseases—convenes its annual Clinical & Scientific Conference to unite physicians, clinical trial investigators, academic researchers, patient advocates, biotech executives, and other key stakeholders for discussion of the most recent advances in neuromuscular disease research. This year's conference took place March 8–11, 2026, in Orlando, Florida.

On Tuesday, March 10, NeurologyLive®’s sister site CGTLive® sat down with Angela Lek, PhD, MDA's chief research officer, to hear her perspective on the conference's standout themes and highlights. Lek identified several priorities that cut across disease areas, starting with muscle regeneration and repair—a subject she noted as pertinent to many of the 300-plus neuromuscular conditions within MDA's scope, with dedicated sessions examining cell therapy strategies aimed at restoring muscle tissue lost to disease or injury. Conditions receiving focused attention this year included limb-girdle muscular dystrophy, titanopathies, infantile-onset forms of muscular dystrophy, and autoimmune neuromuscular diseases. Lek also noted that sessions addressing amyotrophic lateral sclerosis genetics and translational research efforts were among the featured programming.

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.