Advances in Gene Therapy, Neuroregeneration at the Forefront of MDA 2025

Muscular Dystrophy Association (MDA), established in 1950, and is set to convene for its 75th meeting. Looking ahead to this year’s event, Sharon Hesterlee, PhD, chief research officer at MDA, praised the breadth of research to be presented. With presentations, posters, and sessions spanning over 300 diseases, the opportunities to collaborate across specialties, learn, and “cross-pollinate” makes MDA truly special, she noted.

In this interview with The American Journal of Managed Care^® (AJMC^®), Hesterlee discussed the aims of MDA 2025, as well as spotlighted various topics, including a plethora of presentations exploring novel advancements in gene therapy and their capacity to transform the treatment landscape throughout neuromuscular diseases.

MDA 2025 will be held in Dallas, Texas, from March 16 until March 19. To stay up to date on AJMC’s coverage throughout this year’s meeting, please visit our dedicated MDA page.

This transcript has been lightly edited for clarity; captions were auto-generated.

Transcript

What would you say are the goals of this year’s MDA? How do you foresee this year’s meeting building off years prior?

Our medical and scientific conference is really meant to be exactly that: a convergence of clinical and research. The goal is always to bring those groups together and look at that transition in translational research, from bench to bedside. We will have a really good mix of both scientific sessions and clinical medical management sessions together. And I think it builds off previous meetings because this is our 75th anniversary. We're really 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.

Are there any topics you expect to take center stage at MDA 2025? Are there any particular sessions attendees should keep on their radar?

We have a specific focus this year in 1 session on muscle regeneration, and a separate focus in the ALS [amyotrophic lateral sclerosis] track on neuroregeneration too. So I think we're at a point where drugs have been approved and we are able to slow or stop some disease progression, especially in muscle disease. But really, you can't treat muscle that's not there, so I think the next step is to figure out how do we regenerate muscle, which is an organ that regenerates. I don't think it's out of bounds for the nervous system either, which has some regenerative capacity as well. So we have sessions in those areas that I think are of particular interest.

The other thing we're going to do is, we have a whole session that's focused on our MOVR, which is our clinical research registry. So we're looking at a number of different use cases, using MOVR registry data and sort of talking about what we've been able to learn. Particularly since we have so many drugs approved in the space, we'll be taking a look at how those different drugs are being used by the community. So those are 2 areas that will be interesting.

I could go on. We also have a session that I think will be very well-attended on what is happening in the field of gene therapy. Again, now that we have at least 2 drugs approved in this space: zolgensma [onasemnogene abeparvovec] for SMA [spinal muscular atrophy] and elevidys (delandistrogene moxeparvovec-rokl) for Duchenne [muscular dystrophy]. A lot of our clinicians will be talking about the lived experience of actually being able to administer those drugs, and what that experience has been like so far? So I think that'll be a very popular session, and that's a whole track actually.

What are some of the bigger takeaways you hope attendees have after the conference concludes?

I hope they come away with a number of things: an appreciation for the breadth of research that's happening. I mean, because we're an umbrella organization, and we cover multiple neuromuscular diseases, really over 300, I think it's a different take than a single disease research meeting. I hope they come away with new collaborative opportunities and "cross-pollinate" between different areas, and a great appreciation for how much has been accomplished so far. And of course, looking to the future, where we will highlight where there are still needs.