Making an Impact on Day 1 of the MDA Clinical and Scientific Conference

“This is a milestone year,” said Donald S. Wood, PhD, MDA’s President and CEO, as he kicked off the 2025 MDA Clinical & Scientific Conference on March 17, in Dallas. He welcomed more than 2,000 neuromuscular researchers, clinicians, allied healthcare providers, industry professionals, and advocacy organizations attending during MDA’s 75th anniversary year.

Donald S. Wood, PhD, President and CEO of MDA

“Let’s take a moment to reflect on our history, to celebrate the groundbreaking progress we’ve made together, and look ahead to the future we are building,” Dr. Wood said. He emphasized the power of collaboration in building MDA’s legacy and keeping progress moving forward. “MDA’s role as a convener in the neuromuscular space … 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 have and continue to alter the trajectory of diseases like the muscular dystrophies, Friedreich ataxia (FRDA or FA), ALS, myasthenia gravis, and many, many more.”

Dr. Wood then reflected on the current challenges in the neuromuscular field, including threats to biomedical research funding and infrastructure. Meeting these challenges, he said, requires renewed dedication to collaboration. “It is more important than ever that we, the clinicians and researchers doing lifesaving and life-changing work, join with the advocacy community to protect rights and opportunities for the families MDA serves,” he said. “We must continue our work to advance science, innovate, and bring treatments and cures forward. Families are waiting.”

Award winners

For the first time, MDA presented two awards honoring contributions to neuromuscular research — one to a scientist and one to a community member.

Katherine Mathews, MD, recipient of the 2025 MDA Legacy Award for Achievement in Clinical Research

Nora Capocci, Executive Vice President of Healthcare Services at MDA, introduced Katherine Mathews, MD, the 2025 MDA Legacy Award for Achievement in Clinical Research recipient.

Dr. Mathews, a leader in genetic medicine and pediatric neurology at the University of Iowa, is best known for her early work in helping to pinpoint the genetic cause of facioscapulohumeral muscular dystrophy (FSHD), as well as her work in documenting the natural history of Duchenne muscular dystrophy (DMD), the dystroglycanopathies, and FRDA. She has conducted more than 30 industry-sponsored clinical trials focused on neuromuscular diseases, significantly advancing clinical care and scientific understanding. In addition to her research, Dr. Mathews has played a key role in mentoring the next generation of clinical researchers through her leadership as part of the Iowa Wellstone Muscular Dystrophy Specialized Research Center.

Sharon Hesterlee, MDA’s Chief Research Officer, introduced Donavon Decker, who accepted the 2025 MDA Community Impact in Research Award on behalf of himself and his family.

Donavon and four of his siblings were diagnosed with limb-girdle muscular dystrophy (LGMD) type 2D (LGMD2D). Donavon volunteered to participate in the first gene therapy trial for a muscular dystrophy in 1999, which involved having the therapy injected into muscles in his feet. Later, his sister, June Burney was the first person to receive gene therapy via vascular delivery for muscular dystrophy. Their efforts have led to significant improvements in the understanding of LGMD, and his advocacy has inspired others to engage in and support research for rare diseases.

Donavon Decker, recipient of the 2025 MDA Legacy Award for Community Impact

Donavon gave a fascinating account of his experience in the early clinical trial and the relationship he built with Jerry Mendell, MD, of The Ohio State University, who ran the study. Since then, Donavon has continued to work tirelessly to accelerate research efforts and improve the quality of life for individuals affected by LGMD, including testifying in front of Congress for the MD CARE Act in 2001. He also co-founded Angle Therapeutics, which is working to develop non-viral gene therapy for LGMD.

Donavon concluded by giving the scientific audience his perspective on rare disease: “With roughly 30 million Americans that have rare disease, just think of the number of people that know somebody with a rare disease. I would guess that probably 80% to 90% of the people in this country know somebody with a rare disease and would like to see them helped. We need to have clinics and families contact their congressional representatives. The NIH funding for rare diseases is critical. This is not a handout. It returns approximately $2.50 for every dollar investment by the government. It creates thousands of jobs, but better yet, it extends thousands of people’s lives.”

MDA Advocacy wins and goals

MDA Advocacy has also had a milestone year. Paul Melmeyer, MDA’s Executive Vice President of Public Policy and Advocacy, took the stage to talk about significant accomplishments in public policy that MDA hopes to accomplish in the coming year.

He started by recognizing the significant achievements made recently in reforms for accessible air travel, newborn screening for spinal muscular atrophy (SMA) — which is now in every state — and community inclusion for people with disabilities.

Paul Melmeyer, Executive Vice President of Public Policy and Advocacy at MDA

For 2025, key legislative initiatives include:

• Enacting streamlined Medicaid coverage across state lines
• Expanding access to genetic counselors and paid caregivers
• Reauthorizing the US Food and Drug Administration’s (FDA’s) Rare Pediatric Disease Priority Review Voucher Program
• Expanding newborn screening for Pompe disease and DMD

However, Paul expressed concerns about potential threats to some of the advancements the community has made, including lawsuits by airlines to roll back new air travel rules and proposed Medicaid cuts, which would impact access to care and home-based community services for neuromuscular diseases.

“We know that Medicaid is the largest insurer covering those with neuromuscular diseases,” he said. “About 45% of those with neuromuscular diseases are covered by Medicaid.”

Paul urged the audience to go to mda.org/medicaid to share their stories of how cutting Medicaid will affect the neuromuscular community. “Our community is sharing our stories, and we ask for you to do the same,” he said.

Keynote address

Robert Califf, MD, MACC, former FDA Commissioner

Keynote speaker Robert Califf, MD, MACC, has had many milestones in his career. A renowned cardiologist and clinical trial expert, he served as Commissioner of the US Food and Drug Administration (FDA) from 2016-2017 and 2022-2025. He is currently an Instructor in Medicine at Duke University School of Medicine.

Dr. Califf outlined the challenges in biomedical research and healthcare, as well as the opportunities.

Challenges, he noted, include that our healthcare system is fragmented and increasingly driven by a focus on profit. On the drug development side, there currently is not a viable financial and organizational model for manufacturing and distributing treatments for rare diseases. At the same time, biomedical research is losing funding and support from federal agencies.

But Dr. Califf pointed out the silver lining among all the challenges: “This is an opportunity, and it’s people like you who can really make a difference, but you’re going to have to get out there and let your voices be heard.”

Bringing people together

The morning’s keynote session ended with a panel discussion featuring Dr. Carliff, Elizabeth McNally, MD, a researcher at Northwestern University specializing in cardiovascular genetics and inherited heart conditions; Barry Byrn, MD, a physician-scientist at the University of Florida specializing in gene therapy and neuromuscular diseases; David Allison, CEO of TREAT-NMD Services Ltd., an international organization dedicated to advancing neuromuscular disease research and care; and Timothy Miller, MD, Vice President of Enterprise Science and Innovation at ThermoFisher Scientific.

The panel brought together different perspectives on what is currently working well in the neuromuscular disease field, as well as current challenges and solutions.

It was a fitting conclusion to the opening session of the 2025 MDA Clinical & Scientific Conference, which is the largest global gathering of clinicians, researchers, and healthcare professionals from the neuromuscular disease community. For the remaining days of the Conference, participants will attend more than 30 sessions led by some of the world’s foremost experts in neuromuscular disease. They’ll learn about groundbreaking research through the sessions and more than 300 scientific abstracts presented at the Conference. And, they will have opportunities to meet other clinicians and researchers and build new collaborations to keep the momentum of neuromuscular research progress going.