MDA’s Origin Story: Grassroots Legacy

As we celebrate MDA’s 75th anniversary, you may be asking, how did this all begin?

It’s no surprise that MDA started as a grassroots organization. From the earliest days, our mission has been shaped by the stories and needs of people living with neuromuscular diseases and their families.

Founded in 1950

MDA began in 1950 when Paul Cohen, a New York business leader living with facioscapulohumeral muscular dystrophy (FSHD), invited a group of individuals to meet in his office in Rye, New York. Each had a personal connection to muscular dystrophy, and the gathering focused on the urgent need for research seeking treatments and cures. At the time, scientists didn’t understand genetics or the biology behind muscle function, and only one doctor at New York Hospital-Cornell Medical Center was interested in seeing patients with muscular dystrophy: Ade T. Milhorat, MD. Going door-to-door, apartment-to-apartment, the group raised money to award MDA’s first research grant of $1,500 to Dr. Milhorat, a pioneer in muscle disease research.

Building a network of care

MDA’s visionary founders recognized the need for quality neuromuscular disease care and sought to link clinical care and scientific research in a single environment. In 1953, they founded the first MDA Care Centers at NYU Langone Health and University of Rochester Medical Center. These Care Centers brought together clinicians and scientists in collaborative projects to address diagnostic and treatment issues in muscle diseases. This set the stage for what we now know as neuromuscular medicine and gave rise to a network of MDA Care Centers starting in the 1960s.

Today, we know that multidisciplinary care — a treatment approach that involves bringing together healthcare professionals from different disciplines — is the best model of care for people living with neuromuscular diseases because they affect so much more than the muscles. MDA Care Centers were pioneers in this model. MDA Care Centers now serve more than 70,000 unique patients at over 120,000 visits annually.

Scientific breakthroughs

The field of neuromuscular medicine has grown, with more than 300 distinct neuromuscular conditions identified. The turning point in MDA-supported efforts to advance diagnosis and treatment occurred in 1986 with the discovery of the dystrophin gene and its defects that cause Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD). The dystrophin gene remains the largest known gene in the human genome. Its discovery marked a milestone in neuromuscular disease research specifically and in genetic research generally.

The dystrophin gene discovery and the technology that made that discovery possible was developed by Louis Kunkel, PhD, Professor of Genetics and Pediatrics at Boston Children’s Hospital and Harvard Medical School, with funding from MDA.

“The work of Dr. Kunkel and many other MDA-supported clinicians and researchers over the last four decades has opened the frontiers of neuromuscular medicine with some of the first treatments for genetic diseases,” notes Dr. Sharon Hesterlee, MDA’s Chief Research Officer.

Today, MDA has funded more than $1.2 billion in research, and there are more than 20 approved drugs for neuromuscular conditions, including two gene therapies. This March, MDA will host its annual Clinical & Scientific Conference, bringing together around 2,000 researchers and clinicians, allied health professionals, and pharmaceutical professionals working in the neuromuscular medicine field, as well as advocacy organizations and people living with neuromuscular diseases. This meeting helps move the field forward and keeps the individuals and families affected by these conditions at the center of its efforts.

75 years of impact

From the beginning, MDA’s supporters have funded critical research and made MDA’s community programs possible, including MDA Summer Camp. This life-changing week-long overnight camp for youth living with neuromuscular diseases grew from one location in Sussex, New Jersey, with 16 campers in 1955, to thousands of campers and a network of camps across the country today.

Over the years, MDA’s Care Center Network, groundbreaking research, and community initiatives have been shaped by the stories and needs of the families we serve. We have many people to thank for making this possible: people living with neuromuscular diseases, their families, volunteers, researchers and clinicians, donors, corporate partners, and advocates.

In our 75th year, we reflect with gratitude on our lasting impact and how we want to continue to expand access to life-saving therapies, drive momentum in neuromuscular research, meet the changing needs of the people we serve, and empower the neuromuscular disease community.

“MDA has been supporting programs to help people and families with muscular dystrophy and related diseases for 75 years. Over the years, we have helped hundreds of thousands of individuals,” says Donald S. Wood, PhD, President and CEO of MDA. “My goal for MDA is to grow our mission to support more research, advance MDA Care Centers, and to continue to advocate on behalf of patients with neuromuscular diseases so that, ultimately, treatment will be available to all.”