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MDA Announces 2027 Clinical & Scientific Conference Agenda Featuring AI, Gene Therapy, Patient Data and the Latest Neuromuscular Disease Research

Four-day global conference will spotlight emerging therapies, artificial intelligence, genetic medicine, clinical trial innovation and late-breaking research across neuromuscular diseases.

NEW YORK, Monday, October 19, 2026 – The Muscular Dystrophy Association (MDA) today announced the agenda for the 2027 MDA Clinical & Scientific Conference, bringing together researchers, clinicians, health care professionals, industry leaders, advocates and people living with neuromuscular diseases to explore advances in research, therapeutic development and clinical care. Taking place March 21–24, 2027, at Rosen Shingle Creek in Orlando, Florida, the four-day conference will feature five scientific and clinical tracks, keynote and plenary programming, patient and caregiver perspectives, industry forums, poster sessions and opportunities for collaboration across the neuromuscular disease community. The 2027 program will examine how artificial intelligence, gene therapy, genetic medicine, patient data, clinical trial innovation, genetic testing and emerging therapies are reshaping neuromuscular disease research and care. View the full agenda here.

Programming will address diseases and conditions including amyotrophic lateral sclerosis (ALS), Charcot-Marie-Tooth disease (CMT), cardiomyopathies, congenital myopathies, Duchenne muscular dystrophy (DMD), facioscapulohumeral muscular dystrophy (FSHD), Friedreich’s ataxia, immune-mediated neuromuscular diseases, limb-girdle muscular dystrophies (LGMD), periodic paralysis, myasthenia gravis, myotonic dystrophy and spinal muscular atrophy (SMA), among more than 300 neuromuscular diseases and conditions served by MDA.

Key program areas include:

• Artificial intelligence and drug development, including emerging applications of AI in neuromuscular research and therapeutic development;
• Gene therapy and genetic medicine, including advances across DMD, ALS, LGMD and other genetic neuromuscular diseases;
• Patient data and disease registries, exploring how data can strengthen research, clinical trials and understanding of disease progression;
• Clinical trial design and outcome measures, as the therapeutic pipeline continues to expand;
• Newborn screening and genetic testing, with implications for earlier diagnosis and intervention; and
• Therapeutic delivery, muscle regeneration and repair, addressing challenges in translating scientific discoveries into treatments.

Final Day Will Spotlight the Latest Neuromuscular Research
The final day on Wednesday, March 24, is dedicated to the newest and most noteworthy developments in neuromuscular disease research and clinical care. The full-day program, “New, Novel, and Noteworthy: NMD Highlights,” was created in response to the volume and quality of research submissions and will feature focused sessions on Clinical Trial Updates, Molecular Mechanisms, Novel Therapeutics and Late-Breaking Abstracts. The program is expected to feature hundreds of peer-reviewed abstracts, with selected research presented through oral and poster sessions.

“The pace of neuromuscular research and therapeutic development is accelerating rapidly,” said Sharon Hesterlee, PhD, President and CEO of the Muscular Dystrophy Association. “The 2027 agenda reflects how quickly the field is evolving, from artificial intelligence and patient data to gene therapy and innovative clinical trials. We are especially excited about the final day, which will bring together the newest clinical and scientific developments from across the field. The Muscular Dystrophy Association brings the people and organizations driving this progress together to accelerate discovery, strengthen care and help ensure advances reach people living with neuromuscular diseases.”

Watch Dr. Hesterlee’s video here.

Connecting Research, Data and Patient Impact

The conference will also highlight MDA’s MOVR (NeuroMuscular ObserVational Research) initiative, which is evolving into MOVR 2.0, a next-generation, research-driven patient data platform designed to connect health information with research and care. Programming will explore how patient data and disease registries can contribute to a deeper understanding of neuromuscular diseases and support clinical research and treatment development.

Sessions including “From Registry to Ecosystem: Unlocking the Power of Patient Data” will examine the growing role of data in neuromuscular research. Other featured sessions will address evolving therapeutic strategies for Duchenne muscular dystrophy, ALS gene carrier research and innovations in clinical trial design and outcome measures.

“The greatest advances in neuromuscular disease happen when discoveries can move efficiently from research into clinical development and care,” said Angela Lek, PhD, Chief Research Officer, Muscular Dystrophy Association. “This MDA Clinical & Scientific Conference creates a unique environment where discoveries can be shared across disciplines and connected to the people, clinical expertise and systems needed to move them forward.”

MDA currently supports more than 130 active research grants advancing discoveries across neuromuscular diseases. That work is connected to the MDA Care Center Network, where multidisciplinary teams provide specialized care and participate in research and clinical trials.

“The most exciting aspect of this moment in neuromuscular medicine is the growing ability to translate scientific discoveries into therapies that can meaningfully change patients’ lives. The 2027 MDA Clinical & Scientific Conference brings together the scientists, clinicians, industry leaders and advocates needed to move that work forward, from understanding disease biology and developing new therapeutic approaches to designing smarter clinical trials and delivering treatments in the real world,” said Barry Byrne, MD, PhD, Chief Medical Advisor and Board Member at the Muscular Dystrophy Association. Dr. Byrne is also the Associate Chair of Pediatrics and Director of the Powell Gene Therapy Center at the University of Florida.

Advancing Patient and Community Engagement

People living with neuromuscular diseases, families and advocates will have opportunities to engage directly with researchers, clinicians and industry leaders.

• MDA-registered community members may participate virtually at no cost or attend in person at a reduced registration rate by applying here.
• Advocacy organizations focused on neuromuscular conditions may participate by invitation in MDA’s NeuroMuscular Advocacy Collaborative (NMAC). Discussions will address research, clinical trial readiness, access to care and public policy.
• Organizations may reserve a table in the Patient Advocacy Pavilion.

“Creating meaningful opportunities for advocates to engage directly with researchers, clinicians and industry is essential to ensuring that research reflects the priorities and experiences of the neuromuscular community,” said Paul Melmeyer, MPP, Executive Vice President, Public Policy & Advocacy, Muscular Dystrophy Association. “These conversations help ensure that the patient voice is part of the decisions and discussions shaping research, clinical development, access and policy.”

Abstract and Registration Information

• Final abstract submission: December 14, 2026
• Late-breaking abstract submission: January 18–February 18, 2027
• Abstract notifications: January 25, 2027
• Conference embargo lifts: March 21, 2027, at 6 p.m. ET
• Early-bird registration: Through December 31, 2026
• See the Abstract Submission Guidelines for additional information.

Join the Conversation
Follow updates using #MDAconference across MDA social channels.

Sponsors & Partners
MDA extends its sincere appreciation to the sponsors and partners whose support helps bring the neuromuscular community together at the 2027 MDA Clinical & Scientific Conference, including:

• Strength of Life: Biogen, BridgeBio, Dyne Therapeutics, Novartis
• Circle of Strength: argenx, ITF Therapeutics, Sarepta, Scholar Rock
• Media Partners: Bionews, CheckRare, Rare Disease Advisor, NeurologyLive, CGTLive

Media contact: press@mdausa.org

About the MDA Clinical & Scientific Conference

The MDA Clinical & Scientific Conference is a global gathering of the neuromuscular disease community, bringing together researchers, clinicians, health care professionals, advocates, industry leaders and people living with neuromuscular diseases. The 2026 conference drew more than 2,400 attendees from more than 40 countries, featuring hundreds of speakers, posters and scientific presentations alongside industry and patient advocacy programming.

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 over 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, TikTok, LinkedIn, and YouTube.