Orlando, Florida, March 12, 2026 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) concluded its 2026 MDA Clinical & Scientific Conference yesterday, convening over 2,400 attendees from 40 countries to help shape the future of neuromuscular research and care. As the largest global gathering dedicated exclusively to the neuromuscular community, the conference featured seven tracks and 32 sessions, 63 oral presentations from abstracts, 195 expert speakers presenting the latest breakthroughs in gene-targeted therapies, muscle regeneration and repair translational research, and multidisciplinary clinical care. Key highlights included advances in gene editing, RNA-targeted therapies, biomarker development, clinical trial optimization, and evolving models of care—reinforcing MDA’s role as a leading convener driving collaboration across research, clinical care, advocacy, industry, and the broader community. See the full agenda here.
President and CEO, Sharon Hesterlee, PhD, Sets the Vision
Sharon Hesterlee, PhD, President and CEO of MDA, opened the conference emphasizing the defining moment in neuromuscular medicine, where scientific discovery is rapidly translating into approved therapies and new standards of care. “The MDA Clinical & Scientific Conference is where discovery becomes direction,” said Dr. Hesterlee. “Gene-targeted therapies, precision diagnostics, and innovative care models are moving from bench to bedside faster than ever before. Our responsibility — together — is to ensure every breakthrough translates into longer lives, greater independence, and better outcomes for the families we serve.”
Keynote Address: John F. Crowley on the Future of Biotech and Rare Disease Innovation
John Crowley, President and CEO of the Biotechnology Innovation Organization (BIO), delivered the keynote address, “Driving the Future of Innovation, Policy, and Patient Impact.” Drawing from his personal journey as a biotech entrepreneur and father of children diagnosed with Pompe disease, Crowley underscored the power of patient-driven innovation and cross-sector collaboration.
“We are living in an extraordinary era for biotechnology—one where innovation, data-driven insights, and patient partnerships are transforming what’s possible for people living with rare diseases,” said Crowley. “For 75 years, the Muscular Dystrophy Association has embodied that spirit, fueling discovery, accelerating new therapies, and uniting a global community committed to transforming the standard of care.”
Following the keynote, Crowley joined Dr. Hesterlee for a live Q&A on sustaining biotech innovation, regulatory evolution, and ensuring equitable access to emerging therapies.
Later, Amy Comstock Rick, J.D., CDER’s Associate Director for Rare Disease Strategy and the Director of Strategic Coalitions for the U.S. Food and Drug Administration’s (FDA) Rare Disease Innovation Hub joined virtually to provide regulatory insight on advancing neuromuscular treatments. She discussed the FDA’s Rare Disease Innovation Hub and its efforts to accelerate the development of therapies for rare diseases. She highlighted the importance of strengthening collaboration across the agency’s drug and biologics centers while engaging patients, researchers, and industry to advance regulatory science and address the unique challenges of small patient populations. Rick also encouraged the rare disease community to partner with the FDA through workshops and other engagement opportunities to help shape the future of therapeutic development.
MDA National Ambassador Lily Sander, who lives with Charcot-Marie-Tooth disease (CMT), received a standing ovation after sharing her personal journey with the audience of clinicians, researchers, and advocates. In her remarks, Lily described the daily planning required to manage fatigue and conserve energy, offering a powerful reminder of the real-world impact of neuromuscular disease and the importance of advancing research, care, and therapies for the community.
View the following videos:
- John Crowley’s full keynote address here.
- John Crowley’s highlights here.
- Amy Comstock Rick remarks here.
- Lily Sander’s speech here.
- Recap video here.
Honoring Excellence: 2026 MDA Legacy Awards
MDA Legacy Award for Achievement in Clinical Research
Recipient: Michio Hirano, MD, Columbia University Irving Medical Center
Presented by Angela Lek, PhD, Chief Research Officer, MDA
Dr. Hirano, and MDA Care Center Director, was recognized for decades of pioneering work in mitochondrial and neuromuscular disorders, advancing genetic discovery, therapeutic development, and clinical trial readiness across multiple disease areas.
MDA Donavon Decker Community Impact in Research Award Recipient: Allison Moore, Founder & CEO, Hereditary Neuropathy Foundation
Presented by Lily Sander, MDA National Ambassador
Lily Sander, who lives with CMT, presented the award to Allison Moore, also living with CMT, honoring her leadership in elevating patient voice, advancing global registries, and accelerating therapeutic development in inherited neuropathies.
Inaugural MDA Research Momentum Award
Recipient: Łukasz Sznajder, PhD, MSc, Assistant Professor at the University of Nevada, Las Vegas (UNLV)
Presented by Angela Lek, PhD, Chief Research Officer, MDA
MDA presented the first-ever MDA Research Momentum Award to Łukasz Sznajder, PhD, MSc, Assistant Professor at the University of Nevada, Las Vegas (UNLV), recognizing his innovative molecular genetics research advancing understanding of myotonic dystrophy, ALS, and related neuromuscular disorders. The award highlights MDA’s investment in emerging scientific leaders shaping the next generation of neuromuscular discovery.
Scientific and Clinical Impact by the Numbers
- 2423 total attendees
- 2020 in-person
- 403virtual
- 40 Countries represented
- 7 Tracks across 32 sessions
- 195 Speakers
- Over 500 posters
- 63 Oral Abstract presentations
- 50 Exhibitors
- 20 Industry Forums
- 27 Patient Advocacy Pavillion participants
- 31 Advocacy organizations attending NMAC meeting
- 20 Industry Forums
- 27 Patient Advocacy Pavilion participants
Looking Ahead
The 2027 MDA Clinical & Scientific Conference will take place March 21-24, 2027, in Orlando, Forida in a new location at Rosen Shingle Creek. Registration opens this summer at MDAconference.org.
Sponsors & Partners
MDA extends its appreciation to the sponsors and partners whose support helps bring the neuromuscular community together at the 2026 MDA Clinical & Scientific Conference, including:
- Strength of Life: argenx, Biogen, BridgeBio, Novartis
- Circle of Strength: ITF Therapeutics, REGENEXBIO, Scholar Rock
- Impact: Catalyst Pharmaceuticals, Dyne Therapeutics, UCB
- Empowerment: Avidity Biosciences, Sarepta Therapeutics, Edgewise Therapeutics, Genentech, Johnson & Johnson
- Media Partners: Bionews, Rare Disease Advisor, NeurologyLive, CGTLive
Media inquiries: press@mdausa.org
About Muscular Dystrophy Association
Muscular Dystrophy Association (MDA) has been at the center of progress for people living with muscular dystrophy, ALS, and over 300 other neuromuscular conditions for 75 years. We unite researchers, clinicians, advocates, and families to speed the pace of discovery, improve access to expert care, and ensure inclusion in every aspect of life. Our mission is simple: give the people we serve the tools and opportunities to live longer, more independent lives. To learn more, visit mda.org. Follow MDA on social media on Instagram, Facebook, X, Threads, Bluesky, TikTok, LinkedIn, and YouTube.
Attachments
Mary Fiance, National Vice President, Strategic Communications Muscular Dystrophy Association press@mdausa.org
