Regenerating Neurons, Muscle, and Hope in the Field of Muscular Dystrophy

When the Muscular Dystrophy Association (MDA) was founded in 1950, research on this group of disorders was limited—and treatment options even more so.¹ In the years that followed, advocacy efforts by stars like Jerry Lewis drew attention and funding via telethons and awareness campaigns, but cures remained out of reach for decades. Now, as the MDA meets to celebrate its 75th anniversary at its 2025 Clinical & Scientific Conference, the stars of the show will be the researchers and clinicians who have brought the field to an exciting precipice where halting or even reversing the march of disease progression is a real possibility, thanks to advances in novel drugs, gene therapy, and stem cell treatment.

The 2025 meeting, which will be held in Dallas, Texas, from March 16-18, kicks off with opening remarks by MDA President and CEO Donald S. Wood, PhD, followed by research updates from Sharon Hesterlee, PhD, chief research officer at MDA.² Hesterlee told The American Journal of Managed Care^® that the meeting will be “showcasing a lot of the work that MDA has done over the years that culminated in the place we are now with multiple drugs approved.”³

The field of muscular dystrophy care is at an exciting precipice where halting or even reversing the march of disease progression is a real possibility. | Image Credit: © NINENII - stock.adobe.com

That first day will also feature a keynote address by former FDA director Robert Califf, MD, as well as looks ahead at neuromuscular research, public policy, and advocacy. According to MDA meeting organizers, these discussions will touch on potential changes in federal policy and how they could impact research and development, as well as the challenges of integrating cutting-edge therapies into real-world practice.⁴

“The advancements in gene therapy, precision medicine, and patient-centered care represent a bright horizon,” Califf stated in an MDA press release, “but much work remains to ensure equitable access to these groundbreaking therapies and a full understanding of the benefits and risks of these major technological advances for all patients that we must address together.”⁴

After these opening sessions, the agenda features multiple tracks spanning from disease mechanisms to care management to drug development, as well as sessions focused on specific disorders such as amyotrophic lateral sclerosis (ALS), Duchenne muscular dystrophy (DMD), Pompe disease, myasthenia gravis, and spinal muscular atrophy (SMA).² There are also opportunities for attendees to learn more about how neuromuscular disease intersects with other specialties, such as pulmonology and cardiology.

A theme that recurs throughout the agenda is the notion of regeneration, or growing back matter that has been lost to the course of a degenerative disease. This could represent an exciting leap forward for the muscular dystrophy field, now that approved drugs can slow or stop disease progression. One panel will discuss neural regeneration as the next frontier in ALS and spinal cord injury, while another will cover progress in regenerating muscle in patients with DMD and other muscular dystrophies through advances like stem cell therapy.

In addition to these sessions and panel discussions, the agenda allots time for the presentation of abstracts throughout the meeting, both at poster receptions each evening and during a jam-packed lineup of novel and late-breaking research on Wednesday. These data span phases from investigational drug candidates to ongoing long-term trial follow-up, and they range in scope from the molecular level—editing genes and regulating ion channels—all the way to the system-level impact of digital health technologies to track outcomes. The late-breaking abstracts also contain data that could expand options for patients who were previously left out of clinical advances; for instance, research on intrathecal administration of disease-modifying gene therapy could benefit patients who were diagnosed with SMA after the age cutoff for receiving the currently approved gene therapy options.

This breadth of research, Hesterlee said, should enable attendees to “come away with new collaborative opportunities and ‘cross-pollinate’ between different areas, and a great appreciation for how much has been accomplished so far.”

References

1. MDA 75. Muscular Dystrophy Association. Accessed March 13, 2025. https://mda75.org/
2. Conference agenda. 2025 MDA Clinical & Scientific Conference. Accessed March 13, 2025. https://www.mdaconference.org/wp-content/uploads/2025/03/2025-MDA-Conference-Agenda-21.pdf
3. Munz K, Hesterlee S. Advances in gene therapy, neuroregeneration at the forefront of MDA 2025. AJMC^®. March 13, 2025. Accessed March 13, 2025. https://www.ajmc.com/view/advances-in-gene-therapy-neuroregeneration-at-the-forefront-of-mda-2025
4. Robert Califf, MD, MAAC, to deliver keynote address at 2025 MDA Clinical & Scientific Conference. News release. Muscular Dystrophy Association; February 27, 2025. Accessed March 13, 2025. https://www.mda.org/press-releases/2025/robert-califf-md-maac-to-deliver-keynote-address-at-2025-mda-clinical-and-scientific-conference