The Field of Neuromuscular Disease is Entering the Era of Treatment

Barry J Byrne, MD, PhD
Credit: MDA

The Muscular Dystrophy Association 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. A key part of both these goals are the MDA Care Centers: over 150 sites that serve as centers of care for patients and as a network for researchers in the field to collaborate.

In the lead up to this year's MDA Clinical & Scientific Conference, which will be held from March 3-6, 2024, in Orlando, Florida, 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 learn more about MDA Care Centers and the conference itself. Byrne noted that this year’s conference will be particularly exciting as the neuromuscular disease field is now entering the era of treatment thanks to novel disease-modifying options like gene therapy.

CGTLive: Can you give a brief introduction about MDA for context, and then tell us about MDA care centers?

Barry J Byrne, MD, PhD: The MDA serves the whole neuromuscular patient community, particularly through establishing a number of MDA Care Centers across the United States—150 of them in total. This is the primary site of care for many of those affected with neuromuscular disease and also a network which allows us to investigate new therapies which benefit the neuromuscular community. So there are many missions of the MDA: Both excellence in patient care and advancing multidisciplinary care, as well as assisting in discovering new therapies that improve the lives of those living with neuromuscular diseases.

For the clinical community who are unaware, could you describe the MDA Clinical and Scientific Conference, and why doctors and healthcare professionals should be interested in attending?

The annual MDA Clinical & Scientific Conference has really entered now what we are considering the era of treatment. This is really exciting to the patient community and to providers. Everyone will gather in Orlando between March 3rd and 6th for this really comprehensive meeting, which covers the spectrum of early discovery science through clinical trial results. I think there's a lot of excitement in the patient and provider community for this conference where everyone comes together to really determine the best way to improve the lives of those living with neuromuscular diseases.

Is there anything specific you are looking forward to at the upcoming 2024 MDA Conference?

I think there's a lot of excitement about the 2024 conference where colleagues from around the country will be able to have one on one meetings. We will share about experiences and best practices and one of those efforts is underway to establish a gene therapy readiness network as we make new products available for patients with Duchenne muscular dystrophy and other neuromuscular conditions. It's important that the MDA care centers really provide state of the art care for patients with neuromuscular disease. I'm really excited to participate in that part.

One other thing I'd like to share is the opportunity for those that are early in their career and trainees that will have the opportunity to really benefit from this era of therapy that we have available in the neuromuscular community now. So I do encourage those that have an interest in neuromuscular disease to consider attending the annual scientific meeting, learn what's going on, and see what opportunities exist in the field of neuromuscular care.

This transcript has been edited for clarity.

Click here to register for the upcoming 2024 MDA Conference.