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Muscular Dystrophy Association Gathers Global Leaders at Annual Conference

The Largest Convening in the Field of Neuromuscular Disease Research & Care in the World

Bringing together more than 1,200 attendees both in-person and virtually, MDA leads the way as the only voluntary health organization in the neuromuscular disease space to combine the latest in research, clinical care, treatment and management, with the goal of transforming care through innovation and allowing people living with neuromuscular disease to live longer lives.

NEW YORK, NY, Feb. 9, 2022 — The Muscular Dystrophy Association (MDA) announced today the schedule for the 2022 MDA Clinical & Scientific Conference. After two years of pivoting to an innovative virtual event, the organization is convening renowned researchers, clinicians, academicians, advocates, and industry leaders from around the world both in person at the Gaylord Opryland Resort and Convention Center in Nashville, Tennessee, March 13-16, 2022, and via live stream. More than 1,200 neuromuscular clinicians and researchers from the United States and around the world, are expected to gather to hear from more than 120 renowned expert presenters in 29 sessions on the latest clinical care and research advances for more than 40 neuromuscular diseases such as Duchenne muscular dystrophy (DMD), amyotrophic lateral sclerosis (ALS), Charcot-Marie-Tooth (CMT), and spinal muscular atrophy (SMA).

This year’s conference also features 200 abstracts that will take the form of posters or talks that will highlight basic, translational, and clinical research. Day four of the conference will be devoted to oral presentations from selected abstracts submitted by scientific and clinical researchers. In addition, MDA will host the 2nd Annual Insights in Research Investor Summit (IRIS), which will provide an opportunity for companies and inventors in the neuromuscular disease space to showcase investment and licensing opportunities. Outside of the sessions, the Patient Advocacy Pavilion will provide an engaging environment for advocates and industry partners to collaborate in meaningful ways. The full conference agenda is available here.

“MDA’s annual conference is the largest and most important convening on advances in understanding and treating neuromuscular diseases in the world. We bring together scientists and clinicians from around the globe to share their progress, best practices for conducting research, new diagnostic approaches, and information on treatments. As a researcher myself earlier in my career, I am both proud and excited to welcome the attendees of this conference, both in-person and remote, and get the chance to hear about the groundbreaking work they are doing in the field of neuromuscular disease research,” said Donald S. Wood, PhD, president and CEO of MDA.

“The MDA Clinical & Scientific Conference has become a prime destination for academic researchers, pharmaceutical and biotech companies to report on their latest clinical trial findings,” said MDA’s Chief Research Officer, Sharon Hesterlee. “Our conference is the only one in the neuromuscular space that really combines everything from basic research to clinical care, treatment, and management.”

MDA anticipates several high-profile data presentations on Wednesday, March 16, during clinical trial presentations. Among the session highlights are:

• Functional/PROs/Composite Clinical Trial Endpoints in DMD (CME Accredited Session) Erik Henricson, PhD, MPH (Chair)
  Session will explore the uses and intersections between Clinically Reported Outcome tools, Person Reported Outcome tools, and single- and multi-domain composite endpoints in DMD clinical research. Emphasis will be placed on how tools create a continuum of measures that can be extended from observed clinical task performance, to individual perceptions of ability, to community mobility and participation and life satisfaction.
• New Gene Discoveries in NMD (CME Accredited Session) Alan Beggs, PhD (Co-Chair) Angela Lek, PhD (Co-Chair)
  Session will provide an overview of the rich and rapidly evolving body of knowledge in this area, with talks focused on collaborative and crowd-sourcing approaches to information sharing for new disease gene discovery, the latest on genetic heterogeneity among inherited peripheral neuropathies, approaches to undiagnosed neuromuscular diseases, and new genetic modifiers, which can impact the course of a patient’s disease.
• Mitochondrial Interventions: Small Molecule and Gene Therapy Carlos Moraes, PhD (Chair)
  Session will explore a few approaches with great clinical potential that use small molecules or genetic manipulation.
• CRISPR – The Next Generation Eric Olson, PhD (Chair)
  Session will focus on recent advances and future challenges in translating CRISPR gene editing for genetic myopathies from animal models toward human translation.
• Impact of Gene Transfer Therapy on Long-Term Multidisciplinary Care (CME Accredited Session) Emma Ciafaloni, MD (Co-Chair) Jonathan Brandsema, MD (Co-Chair)
  Session will address the impact of newborn screening and gene therapy on the neuromuscular clinics’ infrastructure and on the multidisciplinary teams; new challenges and opportunities for innovation in the multidisciplinary model; new approach to long term management and assessment of new phenotypes in the era of Gene therapies; and how to optimize equitable access to novel gene therapies and multidisciplinary care in the era of telemedicine.
• Progress in Brain Interface Technology Daniel Rubin, MD, PhD (Co-Chair) Leigh Hochberg, MD, PhD (Co-Chair)
  Session will review recent progress, with leaders in the field sharing their vision for the clinical applications and future of these important technologies.

Thank you to our Strength of Life Partners: Genentech & Sarepta; Circle of Strength: Biogen; Impact Sponsors: Amylyx, PTC & Pfizer; Empowerment Sponsors: Apellis & NS Pharma; Champion sponsors: Astella Gene Therapies, BioMarin, Brainstorm Cell Therapeutics, Dyne Therapeutics, Edgewise Therapeutics, Fulcrum Therapeutics, Hillrom, Novartis Gene Therapies, PepGen, RegenxBio, Santhera, Scholar Rock, Stealth Biotherapeutics, Zogenix; Friends of MDA: Alexion AstraZeneca Rare Disease, Harmony Biosciences, Mitsubishi Tanabe Pharma America, Rare Disease Advisor, Reata Pharmaceuticals.

About the Muscular Dystrophy Association

For 70 years, the Muscular Dystrophy Association (MDA) has been committed to transforming the lives of people living with muscular dystrophy, ALS, and related neuromuscular diseases. We do this through innovations in science and innovations in care. As the largest source of funding for neuromuscular disease research outside of the federal government, MDA has committed more than $1 billion since our inception to accelerate the discovery of therapies and cures. Research we have supported is directly linked to life-changing therapies across multiple neuromuscular diseases. MDA's MOVR is the first and only data hub that aggregates clinical, genetic, and patient-reported data for multiple neuromuscular diseases to improve health outcomes and accelerate drug development. MDA supports the largest network of multidisciplinary clinics providing best-in-class care at more than 150 of the nation's top medical institutions. Our Resource Center serves the community with one-on-one specialized support, and we offer educational conferences, events, and materials for families and healthcare providers. MDA Advocacy supports equal access for our community, and each year thousands of children and young adults learn vital life skills and gain independence at summer camp and through recreational programs, at no cost to families. During the COVID-19 pandemic, MDA continues to produce virtual events and programming to support our community when in-person events and activities are not possible. MDA's COVID-19 guidelines and virtual events are posted at mda.org/COVID19. For more information, visit mda.org.