Collective Advocacy, Shared Priorities, and What’s Ahead at MDA 2027

Each spring, the Muscular Dystrophy Association’s (MDA) Clinical and Scientific Conference brings together researchers, clinicians, industry partners, and a broad coalition of patient advocacy organizations spanning the neuromuscular disease community. At the heart of that gathering is the Neuromuscular Advocacy Collaborative, convened by MDA’s public policy and advocacy team, which allows organizations to align on shared priorities and coordinate advocacy efforts for the year ahead. The 2027 conference, scheduled for March 21-24, will serve as the next major convening point for the NMAC.

Paul Melmeyer, MPP, is Executive Vice President of Public Policy and Advocacy at the MDA and has been closely involved in the growth and coordination of the Neuromuscular Advocacy Collaborative over the past several years. He sat down with NeurologyLive ahead of the 2027 conference to discuss how the NMAC has evolved from an annual in-person gathering into a year-round collaborative effort and what that means for the communities it serves.

In this Q&A, Melmeyer explains which advocacy organizations will be represented at MDA 2027, how the NMAC has expanded its reach and frequency of engagement, what the primary goals heading into the conference are, and how he envisions the collaborative continuing to evolve as the neuromuscular landscape shifts with new therapies, newborn screening programs, and changing community needs.

NeurologyLive: Provide some insights on which advocacy groups we can expect to see at MDA 2027 and a little bit of background on their role within this community.

Paul Melmeyer, MPP: We’re very fortunate that at MDA’s Clinical and Scientific Conference, convening again in March of 2027, we’re going to have patient advocacy organizations throughout the neuromuscular disease community present. We’re grateful that organizations serving those with ALS, with Duchenne muscular dystrophy, with limb-girdle muscular dystrophies, and many others all gather at our conference for many reasons.

They attend to learn from the sessions in which we convene experts to speak on relevant topics of scientific or clinical importance. They convene to meet with researchers, clinicians, and biopharmaceutical companies in the field to collaborate and develop relationships and put together priorities for the ensuing year.

Within the Neuromuscular Advocacy Collaborative that MDA’s public policy and advocacy team convenes at the conference, the neuromuscular disease advocacy organizations are able to collaborate on our public policy and advocacy goals for the ensuing year.

How has the NMAC evolved over time, and what are some of the lesser-known benefits these groups bring to advancing research and care for neuromuscular conditions?

We have been very fortunate to convene the Neuromuscular Advocacy Collaborative for several years at this point, and we do hold an in-person annual meeting at the MDA Clinical and Scientific Conference each spring. One of the ways the NMAC has evolved over the last several years is that we are meeting more frequently and working more consistently across the entire year on shared priorities.

Previously, we might meet in the spring and talk about areas in which we hear from our community as priorities for all of us to work on within public policy and advocacy. But now, while we still convene in person and this meeting is critically important to setting up the ensuing year for success, we’re in much more frequent collaboration throughout the year, whether through email or meetings.

What we’re doing now is actually running campaigns together. We are working to ensure our community has access to the treatments and care they require, to ensure that their rights as disabled Americans are not being violated, that they’re still able to access education, employment, transportation, and accessible voting.

With more and more FDA-approved therapies reaching our community, we’re also working to ensure everyone within the neuromuscular disease community is able to not only obtain an FDA-approved therapy, but work toward that FDA approval through biomedical research and the clinical trials that may subsequently happen to test promising therapies for our community. We collaborate on ways to encourage Congress to pass laws to serve our community, and we collaborate on ways to encourage regulatory agencies to take administrative actions in support of our community.

Not only has the Neuromuscular Advocacy Collaborative grown over the last five to ten years with many new members, but we’ve also expanded the areas in which we collaborate and the frequency by which we do so. These organizations are imperative to fostering an environment in which biomedical research is being funded, is happening, and is actually resulting in discoveries for neuromuscular disease patients.

They are also critical to ensuring that our care ecosystem, including at MDA’s multidisciplinary care center network but also throughout the broader care infrastructure, is one where our community is receiving the very best care possible. Without the patient advocacy organizations, I’m not sure where many in the neuromuscular disease community might find themselves.

Heading into MDA 2027, what are some of the primary goals or areas of focus for the NMAC?

One of the beauties of the Neuromuscular Advocacy Collaborative convening at the MDA Clinical and Scientific Conference in 2027 is that we’re really able to zoom in on the issues that are most pressing to the community at that time. One of the ways we spend our time at the conference is comparing what we’re hearing from the different neuromuscular disease communities about what might be most pressing within their experience of navigating a neuromuscular disease.

Perhaps we’re hearing a lot about challenges in accessing care or affordable health insurance, and once having insurance, actually being able to access the medical devices, drugs, or clinicians that should be covered. If that is what we’re hearing from the community, that’s what we’ll be focusing on.

Or perhaps we’re hearing frustrations about biomedical research not advancing as quickly as we all might like, and so we might discuss ways to collaborate to ensure that our biomedical research infrastructure in neuromuscular diseases is well funded and well operating. Or perhaps we’ll hear frustrations about accessibility and accommodations and just being able to live a fully independent life.

We really do keep our ear to the ground throughout the year, and I know all of our organizations bring what they hear from their communities to this meeting, so we can focus on the areas that are going to be most impactful. We’re almost going to have to wait and see for the meeting to know exactly what we’ll focus on, because what our community is focused on in March of 2027 is going to be the topic of conversation.

How do you envision the NMAC continuing to evolve as neuromuscular medicine and care changes over the next several years?

There are many ways in which we envision the Neuromuscular Advocacy Collaborative evolving, because the communities represented within it can oftentimes find themselves in very different situations. There may be communities fortunate enough to have many different FDA-approved therapies, and so they may be focused on the next generation of treatments or on access challenges to existing ones.

Perhaps for these communities, the diagnostic odyssey has been successfully shortened through various means, even including newborn screening, as we see for the Duchenne, SMA, and Pompe communities. For those communities, the focus may be on living a full life with careers, education, and employment, and on how organizations like MDA can be serving them in that next chapter.

On the other hand, also within the Neuromuscular Advocacy Collaborative, there are organizations still working to find the very underpinnings of their disease. There may still be an immense diagnostic odyssey. There may still be so much medical research needed to even discover a first potential FDA-approved therapy. When we gather everyone together in the same room, those organizations and those communities can learn from the communities that have been there.

It isn’t that long ago that the Duchenne community or the SMA community were also striving to get that very first FDA-approved therapy. What we can envision as the NMAC evolves is that because our communities are evolving, advancing in obtaining new therapies and unlocking new clinical approaches, they are learning from each other and taking those learnings to grow within their communities and then collaborate on the next steps. And it’s at the Neuromuscular Advocacy Collaborative convening where that can all happen.

Transcript edited for clarity. For more information about MDA 2027, click here.