MDA 2025 as a Mixer for Strategies and Collaboration
Sharon Hesterlee, PhD, the chief research officer of the Muscular Dystrophy Association discussed the upcoming MDA Clinical and Scientific Conference, which will be held March 16-19, in Dallas, Texas.
Sharon Hesterlee, PhD
The Muscular Dystrophy Association (MDA) holds its Clinical and Scientific Conference every year to gather scientific and medical professionals, patients, and advocacy groups together to discuss and share the latest advances in the field of neuromuscular disorders. For 2025, the conference is being held from March 16 to 19 in Dallas, Texas.
In the lead up to the conference CGTLive®'s sister site, NeurologyLive®, spoke with Sharon Hesterlee, PhD, the chief research officer of the Muscular Dystrophy Association, to get her perspective on what attendees can look forward to at the upcoming meeting. Hesterlee emphasized the diversity of the event's program and highlighted several notable sessions, tracks, and meetings within the conference.
CGTLive: Can you give an overview of what the MDA conference is for anyone who is not familiar with it?
Sharon Hesterlee, PhD: The MDA conference I think is pretty unique because it's a really true blend of science, research, and clinical care because MDA, of course, supports a network of care centers across the United States. We have all of our care center directors here in one place, we have all of our scientific advisors in one place, and so that creates a really rich environment for mixing those 2 areas.
This year, we've got a couple of focus areas that we're excited about. We are specifically focused on muscle regeneration, I think, in recognition of the fact that there has been so much progress with things like gene therapy and exon skipping for Duchenne specifically. We still have people who've lost a significant amount of muscle mass over time, and we know you can't treat muscle that's not there. We have to find a way to regenerate muscle, and muscle does regenerate. So we do have a focus there. We have a whole session at the conference that'll be focused on muscle regeneration, sort of building off a meeting that we had earlier this year. I think that's a really exciting new direction that we're going to see more and more activity around.
As usual, we have a whole day-long track on amyotrophic lateral sclerosis (ALS) that will talk about the role of glial cells, brain-computer interface, and a section on biomarkers and genetics, and all of these areas that have continued to evolve and contribute to our understanding of ALS. I think that the speaker lineup is going to be great for that one.
We also, in addition, have some focus on specific disease areas this year. There's going to be [focus on] myotonic dystrophy and ocular pharyngeal muscular dystrophy—which is one that you don't hear a lot about, but it affects people generally who are older, but it causes some significant disability over time and there's some really interesting new strategies for treating that disease. I would say stay tuned for that one for sure.
On the clinical side, we'll focus on multidisciplinary teams and how you help build those teams, and also training. Training is a really big issue. We don't have enough neuromuscular specialists in the neurology field, so we'll talk about training and also diversity within that training. How do we have more diverse providers? We worry a lot about clinical trials reflecting the diversity of the United States and treatment disparities. We also need to have a diverse workforce. We'll have a great session on that, and also one that's intriguingly named, "Changing Policies and Pushing Boundaries". I think you have to attend the meeting and see what that one is about.
That's just a few highlights, an overview snapshot. And of course, as usual, we will have some auxiliary meetings as well. Our care center directors have a meeting where they all get together and are able to discuss things that impact their areas and their care centers. We also have an allied health professionals meeting that takes place beforehand: genetic counselors, physical therapists, and some issues that are really important to that group. We have an advocacy meeting that's usually attended by between 20 and 30 different advocacy groups who work in the neuromuscular space, and they talk about a lot of important issues that are relevant across all of these groups. And finally, we also have a meeting for our trainees, our new up-and-coming development grantees.
So it's a very rich meeting. I think it'll be great this year. Of course, we're coming into our 75th year as an organization, so that's really exciting at this point. The legacy is pretty extensive, and from the standpoint of impact, having invested over a billion dollars in drug development, it's very gratifying to see that there are now many drugs approved and more on the horizon. I should mention also that on Wednesday, we have our late breaking research, and we have a lot of clinical trial readouts. I know I saw this morning that Capricorn has just filed for a biologics license application for their drug for Duchenne. So I think we're going to hear a lot of exciting news on Wednesday too about new drugs in development. All of that comes together, I think, and it's just incredible momentum.
What are your primary goals for the conference?
Our goal is really about community. It's bringing together all these different facets of our community, from clinical care to research. We have companies who are developing drugs who will be present. We have community members represented and many different disease advocacy groups will be at the conference. It's a real melting pot, and I think it's great for fomenting those new collaborations, getting everyone on the same page, and really creating a lot of energy going forward.
Is there anything else you would like to share?
I do want to remind people that if you can't attend in person, that we do have a virtual option as well, so we'll maintain that virtual stream with very rich content, as well.
This transcript has been edited for clarity.
Click here to register for the upcoming 2025 MDA Conference.
