Inside the 2026 MDA Conference: Emerging Research Themes in Neuromuscular Medicine

Each year, the Muscular Dystrophy Association (MDA) Annual Clinical and Scientific Conference serves as a key forum for clinicians, researchers, and allied health professionals to assess where neuromuscular medicine is headed next. This year’s event takes place March 8-11 at the Hilton Orlando hotel in Orlando, Florida. As therapeutic development accelerates across genetic, inflammatory, and degenerative neuromuscular diseases, the meeting has increasingly become a venue for discussing not only trial data, but also the translational, regulatory, and care delivery challenges shaping real-world practice.

Ahead of the release of the 2026 meeting agenda, NeurologyLive® sat down with Angela Lek, PhD, chief research officer at MDA and a program consultant for the NIH Somatic Cell Genome Editing Program, to discuss how this year’s conference was designed. In the conversation, Lek outlined emerging scientific themes, highlighted underexplored research priorities such as muscle regeneration, and explained why the 2026 meeting is positioned as a must-attend event for clinicians navigating rapid change in neuromuscular care.

NeurologyLive: If you want to first just talk a little bit about the agenda itself, what goes into the 2026 annual meeting agenda, and anything you would like to call out for a clinical audience?

Angela Lek, PhD: As you know, our meeting attracts a wide range of stakeholders across the neuromuscular disease community. That includes clinicians, researchers, allied health professionals, industry representatives, and patient advocates. Because of that, we design parallel tracks that cover a broad range of topics to cater to this diverse audience.

The topics are chosen in collaboration with our advisors and through polling the neuromuscular community to understand what timely issues people want to hear more about. The agenda is composed of several tracks that occur in parallel. These include an allied health considerations track, a latest-in-care trends track, research-focused sessions on disease mechanisms and therapeutic strategies, drug development considerations, and a lab-to-life track where we spotlight specific diseases or disease categories and move from bench science to the clinic. We also have a specialized ALS track and a general neurology track.

From a new or emerging concept standpoint, are there topics that may have appeared last year but are being emphasized more in 2026?

The 2026 agenda introduces some fresh topics that the community is really craving. One is nonviral delivery for gene therapy, which is an area of intense interest. Another is immune response considerations, which is a very timely topic in gene therapy, particularly for neuromuscular diseases.

We are also emphasizing next-generation patient registries and infrastructure to strengthen clinical research and data collection. In addition, we are continuing the momentum established this past year around how to regenerate and repair muscle lost to disease. There is also a strong focus on navigating today’s drug development landscape, including regulatory and financial bottlenecks and how to get therapies to patients faster.

We are also introducing more interactive formats, such as Stop the Fellows case challenges and office hours focused on the business of care, which give attendees new ways to learn and connect.

From a community perspective, what types of roles and specialists do you see as most relevant for this meeting?

We really try to bring together all stakeholders who can help drive the field forward. That includes clinicians who provide specialized and multidisciplinary care, researchers applying new technologies to important scientific questions, and patient advocates whose voices are essential.

The meeting is very full, and we also offer specialized networking sessions and workshops. These include a care center directors meeting, allied health workshops and training sessions, networking opportunities, and the neuromuscular advocacy collaborative. These are in addition to the scientific sessions and poster presentations, which allow for exciting and in-depth conversations.

From a research standpoint in particular, we are very excited about this year’s agenda. It highlights innovation across next-generation genetic therapies, disease-modifying drugs, and transformative approaches to clinical care. We have made a concerted effort to select research topics that are broadly applicable across multiple neuromuscular diseases, such as inflammation and immune dysregulation, muscle regeneration and repair, and therapeutic strategies that target classes of diseases like repeat expansion disorders or inherited peripheral neuropathies.

From a clinical research perspective, what areas is MDA prioritizing, especially those that may not have received as much attention in recent years?

One of our strategic priorities is elevating strategies to overcome muscle wasting that occurs as a result of disease. Gene therapy, particularly gene replacement therapy, has been transformative, but the field has largely coalesced around the idea that gene therapy can at best halt disease progression.

We now need to push forward and address how to regenerate and repair muscle that has already been lost. In 2024, we assembled a summit of key opinion leaders to identify gaps and bottlenecks in this space. This included discussions around stem cells and regeneration, current delays in the field, and how we can help move this area forward.

We summarized those discussions in a publicly available meeting report and issued a call for grants to address these gaps. We received hundreds of applications globally. One proposal that rose to the top was led by a researcher in Copenhagen, Abigail Mackey. While it has not yet been publicly announced, MDA and several other nonprofit organizations are coming together to fund this muscle regeneration initiative.

Muscle regeneration is a shared research priority across many neuromuscular conditions, not something limited to a single disease. That is why we reached out to advocacy partners to rally behind this strategy. We will continue this conversation at the 2026 meeting in a dedicated session on muscle regeneration and repair, chaired by Noah Weisleder from the University of Kentucky.

What is your call to action for the clinical community? Why should clinicians attend the 2026 MDA Annual Meeting?

The 2026 MDA Annual Clinical and Scientific Conference is the best place to stay ahead of rapid changes in neuromuscular medicine. From cutting-edge treatments to evolving care standards, attendees will gain actionable insights they can bring directly back to their clinics to improve diagnosis, management, and patient support.

It is also where some of the most cutting-edge research and technologies will be shared. A significant amount of unpublished data will be presented in both oral sessions and posters. Finally, the latest clinical trial readouts will be presented on the last day of the meeting, which is not to be missed.

Transcript edited for clarity.

For more information about registration to the 2026 MDA Conference, click here.