Gene Therapy Will Be a Major Area of Interest for Attendees at MDA’s 2025 Conference

Paul Melmeyer, MPP

The Muscular Dystrophy Association (MDA) will be holding its next Clinical & Scientific Conference on March 16-19, 2025, in Dallas, Texas. In the anticipation of this, CGTLive® reached out to Paul Melmeyer, MPP, the executive vice president of public policy & advocacy at MDA, to get his view on what to look forward to at the upcoming meeting.

Melmeyer explained what MDA and the conference are and how they serve the neuoromuscular disease community. He then noted that gene therapy will likely be an important topic of interest at the 2025 conference for all stakeholders who attend, and explained what he is looking forward to at the meeting.

CGTLive: Can you give a brief introduction about what MDA is?

Paul Melmeyer, MPP: We at MDA serve the neuromuscular disease community through a variety of different programs and initiatives, including our community programs that bring summer camps, family getaways, and education to our community. We are there for our community as they navigate their healthcare journey through our over 150 care centers. We in our public policy and advocacy effort try to change the laws and work with policymakers to improve the lives of those we serve. And through our research efforts, we try to accelerate the developments of new treatments, cures, and interventions for the neuromuscular disease community.

Could you describe the MDA Clinical and Scientific Conference, and why doctors and healthcare professionals should be interested in attending?

At MDA's Clinical and Scientific Conference, we convene the entire stakeholder community within the neuromuscular disease field. That, of course, primarily can include those researchers and clinicians that every day are working to improve the health of the neuromuscular disease patient community. But also at our conference, we convene the patient advocacy organizations—not just MDA, but 30 to 40 different patient advocacy organizations who also serve the neuromuscular disease community. With them, we actually hold a meeting called the Neuromuscular Advocacy Collaborative, where we all get together and talk about what we want to prioritize in public policymaking in our advocacy for the ensuing year. Not only do those organizations participate within that meeting, but they also participate within the patient advocacy pavilion, within the exhibit hall at the conference. It's there that the clinicians and the researchers who are attending our conference can actually meet with the patient advocacy organizations, get to know them, get to know their initiatives, and find new ways to be working with them. For the clinicians who are considering attending MDA's Clinical and Scientific Conference, not only do they get to meet their fellow clinicians and the researchers within the space, but they also get to meet the many patient advocacy organizations in this space, as well as patients, and potentially even policymakers as well.

Since the last MDA conference, have there been any significant advances in gene therapy for neuromuscular disorders that you expect will be a focus at the upcoming conference?

The field is learning at lightspeed pace about gene therapies—and by the field, I mean really everybody. From the research community, we've seen some immense advancements in delivery mechanisms, as well as other efforts to bring gene therapies to communities that do not yet have a gene therapy approved for that specific neuromuscular condition.

For the clinical community, the clinicians now within the neuromuscular space have had greater experience with the 2 gene therapies that have been approved by the FDA. With over a year of experience at this point [for Sarepta's Duchenne muscular dystrophy gene therapy], including with a broader number of individuals who qualify to take the gene therapy, it's really quite an amazing time to be a clinician in neuromuscular diseases, learning more about how to administer gene therapies and how these gene therapies that are approved by the FDA actually work for those with neuromuscular conditions.

For those within the patient advocacy community who will be joining, many of the organizations who will be present at our conference serve those communities that are actively attempting to access gene therapies, many of whom have been successful.

And the many other communities that will also be joining us at the conference, by and large, are all hoping that a gene therapy, if not already approved for their condition, will be approved for their condition within the next handful of years.

So really, for everybody who will be attending the conference in March, gene therapy will be a relevant topic and a relevant conversation for them to join.

Is there anything specific you are looking forward to at the upcoming 2025 MDA Conference?

I'm really looking forward to the increased number of patient advocacy organizations who will be joining MDA'S upcoming conference. Really it underscores what we're going for within our 75th year as an organization. We're nearly 75 years old, and really what we are looking for is to emphasize the legacy and the impact we as an organization have made, but [also] the momentum that we have going forward. I think there's actually no better place to see that momentum than in the way in which we're able to convene the patient advocacy community within our conference, to be able to have everybody across the neuromuscular disease spectrum convened in one place, talking with each other about how we can better serve the community that we all collectively serve. I've only seen that conversation grow over the years in which we've been convening the advocacy community at our conference for the last couple of years. Each year our meeting has gotten bigger, each year the patient advocacy pavilion has gotten bigger, and each year more community leaders join us at the conference. In our 75th year, it's only going to be bigger than what we've had previously. That's what I'm most excited to see at our conference, and see the momentum that it launches from there.

This transcript has been edited for clarity.

Click here to register for the upcoming 2025 MDA Conference.