MDA’s Upcoming 2025 Clinical & Scientific Conference

Barry J Byrne, MD, PhD
Credit: MDA

The Muscular Dystrophy Association (MDA) is an organization dedicated to both providing state-of-the-art care for patients with neuromuscular diseases and to advancing research related to the development of new therapies for neuromuscular disease indications. Each year, the organization holds the MDA Clinical & Scientific Conference to bring together various experts and stakeholders to share and discuss the latest findings in neuromuscular disease research.

In the lead up to this year's meeting, which will be from March 16 to 19, 2025, in Dallas, Texas, CGTLive™ sat down with Barry J Byrne, MD, PhD, the chief medical advisor of MDA and a physician-scientist at the University of Florida, to get his perspective on what to expect at the conference and what he is most looking forward to this time around. Byrne highlight several topics that will be discussed at the 2025 meeting, including maximization of muscle regeneration in patients being treated for neuromuscular diseases and real-world evidence seen with delandistrogene moxeparvovec-rokl (Elevidys; Sarepta Therapeutics) in patients with Duchenne muscular dystrophy (DMD).

CGTLive: Can you give a brief introduction to the MDA Clinical and Scientific Conference for those who are not aware?

Barry J Byrne, MD, PhD: The MDA Clinical and Scientific Conference will be held this March in Dallas. This meeting is really an opportunity for all the professionals that are involved in the care of patients with neuromuscular disease to come together to exchange ideas and best practice concepts that help improve the lives of patients living with neuromuscular diseases.

What do you expect to be some of the major themes at the 2025 conference?

Last year, we had almost 1,500 attendees. We expect the same attendance or greater this year in Dallas. Of course, this is an important milestone for MDA. This conference marks the 75th year of the formation of MDA and really 75 years of of the commitment to caring for and studying ways to improve the lives of people living with neuromuscular diseases. Really, this is the era of therapies for neuromuscular diseases. In the past year, we saw an additional few strategies that are transformative in the care of boys with DMD and we have continued to make advances in spinal muscular atrophy care following on the enormous success of the release of Zolgensma in 2019. There are now more than 3,000 patients who've received that gene therapy product, and we'll see more new ideas about gene therapy strategies for both central nervous system (CNS) disorders, as well as muscle disorders that affect this population. [We're] excited to understand what new advances are on the horizon from the meeting.

Is there anything different about the conference this year in comparison to previous years?

One of the themes in 2025 is really to understand how muscle regeneration can be maximized in patients with degenerative neuromuscular diseases. This is obviously important to those with later stage conditions. We'll now have a new window on how to predict the rate of change in patients using new outcome measures that have been developed in clinical trials and will be hopefully available in clinical practice. We now also have newborn screening for DMD in several states, with the expectation that will expand in 2025. That provides an opportunity for early treatment and hopefully better outcomes.

What are you personally most looking forward to at the conference this year? Are there any specific presentations or tracks that interest you most?

We will have several sessions related to real-world experiences with the use of the expanded-access to Elevidys, a treatment for DMD in all ages. We're starting to gain experience in the nonambulant population, as well as adults with Duchenne. This will be important to see how this kind of treatment can impact those in later stages of disease. We'll learn more about the expectations for advancing cardiac care in the nonambulant population, where that matters significantly to them as a principal disease symptom that affects longevity.

That, plus some challenges we face in the care delivery model—access to therapy—understanding the unique aspects of reimbursement and management of patients that opt for treatment. That'll be a session where we'll be discussing the best approach to make sure that the widest community of patients has access to current therapies and future therapies.

This transcript has been edited for clarity.

Click here to register for the upcoming 2025 MDA Conference.