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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 ...
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Muscular Dystrophy Association Celebrates FDA Approval of Sarepta Therapeutics' ELEVIDYS for Treatment of Duchenne Muscular Dystrophy

The FDA approval of the first gene therapy for Duchenne Muscular Dystrophy (DMD) by Sarepta Therapeutics is ground-breaking and designed to target the underlying cause of the disease. The Muscular ...
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Muscular Dystrophy Association and Coalition to Cure Calpain 3 Partner to Fund $300,000 Research Grant for Gene Therapy Development in Limb-Girdle Muscular Dystrophy

Grant Awarded to Melissa Spencer, PhD, Co-Director of the Center for Duchenne Muscular Dystrophy at UCLA. New York – Tuesday, December 17, 2024 — The Muscular Dystrophy Association (MDA) and Coalition ...
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Duchenne Muscular Dystrophy (DMD)

Until the 1980s, little was known about the cause of any of the forms of muscular dystrophy. In 1986, MDA-supported researchers identified a gene on the X chromosome that, when flawed (mutated), ...
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Duchenne Muscular Dystrophy (DMD)

In Duchenne muscular dystrophy (DMD), muscle weakness selectively affects proximal muscles (close to the trunk) before distal (away from the trunk) muscles, and the legs before the arms. Additionally, ...
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MDA Peer Connections

We understand the importance of connecting with others who are facing similar challenges. With MDA’s Peer Connections program, we can connect you with someone with a similar diagnosis to share ...
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FDA Approves Isembyld (apitegromab-mstn), the First and Only Muscle-Targeted Therapy for Spinal Muscular Atrophy, Marking a New Milestone in SMA Treatment

New York, Friday, September 11, 2026 – The Muscular Dystrophy Association (MDA) marks another milestone for the neuromuscular community with the U.S. Food and Drug Administration (FDA) approval of ...
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Jerry Lewis, Longtime MDA National Chairman, Telethon Star and Humanitarian, Dies

CHICAGO, August 20, 2017 — The Muscular Dystrophy Association is deeply saddened by the death of beloved comedian, performer, humanitarian and former MDA telethon star and national chairman Jerry ...
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FDA Approves Expanded Use of Eculizumab (Soliris) for Pediatric Myasthenia Gravis, Bringing New Treatment Options for Children

New York, March 10, 2025 – The Muscular Dystrophy Association (MDA) welcomes the U.S. Food and Drug Administration (FDA) approval of the expanded indication of Alexion/AstraZeneca’s eculizumab ...
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MDA Community Support Groups

You don’t have to navigate your neuromuscular disease journey alone. As a member of the MDA Community, one of the many ways you can connect with others and share your personal story is by joining an ...
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Congenital Myopathies

The word myopathy means “disease of muscle.” More specifically, myopathies are diseases that cause problems with the tone and contraction of skeletal muscles (muscles that control voluntary movements.