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2025 MDA Clinical & Scientific Conference Showcased Groundbreaking Advances in Neuromuscular Disease Research and Care & Celebrated MDA’s 75th Anniversary

New York, Thursday, March 20, 2025 – The Muscular Dystrophy Association (MDA) concluded its 2025 MDA Clinical & Scientific Conference, convening global leaders in scientific research and clinical care to advance treatments for the neuromuscular disease community. With over 2,500 attendees in-person and online, the conference brought together experts, industry leaders, and patient advocates from 41 countries to discuss cutting-edge developments. This year’s conference was especially significant as it honored MDA’s 75th anniversary, and the organization’s legacy, impact and forward momentum.

Donald S. Wood, PhD, President and CEO of MDA, Opens with a Vision for the Future

Dr. Donald S. Wood welcomed attendees with an inspiring message about the progress in neuromuscular disease research and the critical role of collaboration across disciplines. He said, “The MDA Clinical & Scientific Conference is where breakthroughs happen—where ideas turn into therapies and hope becomes action. As we celebrate MDA’s 75th anniversary, we remain steadfast in our commitment to accelerating treatments, improving patient outcomes, and fostering an inclusive research and care community. Muscular Dystrophy Association’s role as a convener in the neuromuscular space is not just symbolic; it is essential. Together, with you and with our broad and diverse community of MDA families, we create spaces where ideas become action, where research moves from theory to therapy, and where the lives of the people we serve are changed forever as we alter the trajectory of over 300 neuromuscular diseases.”

Keynote Address by former FDA Commissioner Robert Califf, MD, MACC

Dr. Robert Califf, former Commissioner of the U.S. Food and Drug Administration (FDA), delivered the keynote address, emphasizing the importance of regulatory science in advancing neuromuscular disease treatments. He highlighted recent FDA initiatives to support rare disease drug development and the evolving role of patient-centered research in shaping future therapies.

Key takeaways from Dr. Califf:

• In 75 years, we've gone from knowing very little about neuromuscular disease to knowing that there's a genetic basis to understanding how to edit genes and to knock gene function up and down to affect the diseases that affect quality and length of life in neuromuscular disease patients.
• Science funding is in peril right now, and the healthcare delivery system is under tremendous financial pressure with the threat of reduction in funding for programs such as Medicaid, which are so important to this community.
• A viable solution to these challenges is advocacy. The Muscular Dystrophy Association is a critical organization, the big tent, bringing together all these different people with different forms of neuromuscular disease to advocate for both research funding, but also for excellent clinical patient care.”
• Watch the full Keynote Speech here, and highlights here.

Panel Discussion

Following the speech Dr. Califf joined a panel of experts moderated by Sharon Hesterlee, PhD, Chief Research Officer at MDA, with MDA Board members Barry J. Byrne, MD, PhD, University of Florida; Elizabeth McNally, MD, PhD, Northwestern University; David Allison, CEO of TREAT-NMD; and Dr. Timothy Miller, Thermo Fisher Scientific. The panel addressed the critical need for our collective community to continue advocating together to continue the progress made over the past 75 years.

NeuroMuscular Advocacy Collaborative Meeting

The NeuroMuscular Advocacy Collaborative Meeting led by Paul Melmeyer, EVP, Public Policy and Advocacy at MDA included 37 organizations in the neuromuscular disease space. The meeting focused on protecting Medicaid, defending the neuromuscular research ecosystem, and enhancing caregiving coverage and support. The community was encouraged to go to MDA.org/Medicaid to contact their representatives and share their stories. More updates from MDA Advocacy will be provided at MDA.org/Advocacy.

Honoring Excellence: MDA Legacy Award Recipients

Dr. Katherine Mathews received the 2025 MDA Legacy Award for Achievement in Clinical Research for her significant contributions to the understanding and treatment of genetic disorders affecting the neuromuscular system. As a professor of Pediatrics and Neurology at the University of Iowa’s Roy J. and Lucille A. Carver College of Medicine, Dr. Mathews has been a leader in clinical research for over two decades. Her work spans multiple neuromuscular diseases, with a particular focus on Duchenne muscular dystrophy (DMD), facioscapulohumeral dystrophy (FSHD), Friedreich’s ataxia and Limb-girdle muscular dystrophy (LGMD).

Donavon Decker was recognized with the 2025 MDA Legacy Award for Community Impact in Research for his outstanding advocacy and personal dedication to advancing research in limb-girdle muscular dystrophy (LGMD). Diagnosed with LGMD 47 years ago, Decker has become a powerful voice in the community, encouraging participation in clinical trials, supporting research funding, and raising awareness about the impact of this rare disease. He is one of eight siblings, five of whom were diagnosed with LGMD type 2D (LGMD2D), including Donavon, and three of whom have since passed away. Additionally, two of his nieces live with LGMD2i.

As part of the anniversary celebrations, also MDA welcomed special guest Chris Lewis, son of legendary MDA Telethon host Jerry Lewis, who shared heartfelt remarks about his father’s enduring legacy and the impact of MDA’s mission.

Scientific, Clinical, and Advocacy Impact by the Numbers

• 8 specialized tracks covering the latest advancements in neuromuscular disease research and treatment.
• 39 sessions led by world-renowned experts.
• 263 in-person posters
• 379 virtual posters
• 56 oral poster presentations
• 207 speakers from leading institutions and organizations.
• 56 exhibitors, featuring the latest innovations in diagnostics, therapeutics, and patient support services.
• 20 Patient Advocacy Pavilion participants, fostering collaboration between patients, researchers, and industry partners.
• 37 advocacy organizations participated in the Neuromuscular Advocacy Collaborative (NMAC) meeting, reinforcing the importance of policy and access initiatives.
• 18 industry-sponsored forums, providing in-depth discussions on emerging treatments and care strategies.

Looking Ahead

The 2025 MDA Clinical & Scientific Conference reaffirmed MDA’s role as a leader in neuromuscular disease research, care, and advocacy. With groundbreaking science, robust discussions in genetic medicine, muscle regeneration, and a shared commitment to improving the lives of individuals and families affected by neuromuscular diseases. The momentum from this conference will drive continued progress throughout the year.

The 2026 MDA Clinical & Scientific Conference will be held in Orlando, Florida, March 8-11. Registration will be open this summer. For more continued updates and information visit MDAconference.org.

Media inquiries contact press@mdausa.org.

About Muscular Dystrophy Association

Muscular Dystrophy Association (MDA) is the #1 voluntary health organization in the United States for people living with muscular dystrophy, ALS, and over 300 other neuromuscular conditions. For 75 years, MDA has led the way in accelerating research, advancing care, and advocating support and inclusion of families living with neuromuscular disease. MDA's mission is to empower the people we serve to live longer, more independent lives. To learn more visit mda.org and follow MDA on Instagram, Facebook, X, Threads, TikTok, LinkedIn, and YouTube

About Muscular Dystrophy Association’s 75^th Anniversary

In 2025, the Muscular Dystrophy Association proudly marks 75 years legacy, impact and momentum in the fight against neuromuscular diseases. Since our founding, MDA has been at the forefront of research breakthroughs, providing access to comprehensive care, and championing the rights of people living with muscular dystrophy, ALS, and over 300 other neuromuscular diseases. This milestone has been made possible by generations of dedicated support from people living with neuromuscular disease, their families, researchers, clinicians, volunteers, and donors—who boldly drive our mission forward. As we look ahead, we remain committed to honoring this legacy, building on the impact we’ve made together, and continuing our momentum toward transformative progress for people living with neuromuscular disorders. For more information visit MDA75.org.