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Muscular Dystrophy Association Launches Rare Disease Day Campaign to Raise Awareness & Support for Neuromuscular Diseases throughout February

The rare disease community collaborates to raise awareness on Rare Disease Day, February 29.

New York, January 31, 2024 – The Muscular Dystrophy Association (MDA) is proud to announce its Rare Disease Day campaign, shedding light on innovations in research, care and advocacy advanced by the organization to resolve the challenges faced by people living with neuromuscular disease.

Rare Disease Day, observed worldwide this year on February 29, is intended to prompt action by advocating for improved healthcare, increased research, and enhanced support for individuals with rare diseases, fostering a global community committed to making a tangible impact. With over 10,000 rare diseases affecting more than 30 million Americans, MDA's focus is on muscular dystrophy, Amyotrophic Lateral Sclerosis (ALS, also known as Lou Gehrig’s disease), and related neuromuscular diseases, which present unique challenges in diagnosis, care, and research.

"For over 70 years, MDA has relentlessly pushed the boundaries of research in the rare neuromuscular disease space. Since leading the Genetic Task Force that discovered the Duchenne muscular dystrophy gene, MDA has been instrumental in paving the way for targeted treatment innovations. The creation of the NeuroMuscular ObserVational Research (MOVR) Data Hub marks another leap forward, revolutionizing our understanding of NMDs, enhancing patient outcomes, and propelling the development of groundbreaking treatments. MDA’s nationwide network of Care Centers ensures that people living with neuromuscular disease receive specialized, advanced care from medical professionals who are experts in the treatment of rare diseases. These are more than initiatives; they are real-world solutions impacting lives every day,” said Donald S. Wood, President and CEO, MDA.

Community Education

In support of Rare Disease Day, MDA will host a Community Education Webinar on February 29 from 4-5pm ET focused on oculopharyngeal muscular dystrophy (OPMD), one of the rare diseases under MDA’s umbrella. Held in partnership with the OPMD Association, this webinar will provide an overview of OPMD including current research and treatment approaches. The presenter will be Dr. Bernard Brais, Neurogeneticist at McGill University. Registration is free here.

Research and Care

Diversity and rarity of neuromuscular diseases pose difficulties in access to prompt diagnoses, access to treatments and access to care. For almost 75 years, MDA has been the convener in all areas of support for families living with these diseases. From pioneering research that has led to the innovations in gene therapy, to the MDA Care Center Network with multidisciplinary medical care teams at over 150 locations nationwide, to the MDA MOVR data hub connecting people to clinical trials faster than ever before. MDA also provides support for families and professionals through educational programming, support groups, and award-winning MDA Resource Center and Gene Therapy Support Network, so no one diagnosed with a neuromuscular disease goes through their journey alone.

Support

Contact the MDA Resource Center and Gene Therapy Support Network by phone: 1-833-ASK-MDA1 (1-833-275-6321) or email ResourceCenter@mdausa.org.

Advocacy

MDA’s Advocacy work focuses on support for accelerated approvals and novel research, access to healthcare, treatments, independent living, newborn screening, accessible air travel and more. This mission plays a vital role in shaping laws and policies that support the needs of people living with rare neuromuscular diseases. MDA’s work has been key in amplifying the voices of patients and securing crucial victories for our community. MDA is eliminating barriers, promoting independence, and calling on Congress for research funds and policies that empower people to live stronger and on their own terms.

Newborn Screening

“MDA’s advocacy for newborn screening to identify and treat rare diseases early in life is now a reality in all 50 states for spinal muscular atrophy. In addition, we’re increasing adoption of newborn screening for Pompe disease across the country, and we expect to see the first screenings to begin for Duchenne muscular dystrophy in Ohio, New York, and Minnesota. Now that we have had 20+ new FDA treatments approved just since 2015, there can be immediate treatment in some cases that slows progression of muscle disease. Anyone can join our efforts to increase access to lifesaving newborn screening by signing up to be a grassroots advocate at MDA.org/Advocacy,” said Paul Melmeyer, VP Public Policy and Advocacy, MDA.

Rare neuromuscular diseases present complex challenges for the medical and research communities. Limited understanding and resources have traditionally created obstacles for what could be achieved. Thanks to the support of generous partners and funders, including the iconic MDA Shamrocks fundraising campaign, MDA meets these challenges head-on. MDA’s mission work has led to historic advancements in genetic research, improved diagnostic methods, and the development of groundbreaking therapies that once seemed beyond reach.

Fundraising

Throughout February and March, for 42 years, the iconic MDA Shamrocks campaign will be available in participating retailers nationwide, and online. Online donation of $35 or more in February and March, include a special limited-edition t-shirt (while supplies last) with the iconic MDA.org/Shamrocks2024, designed by MDA advocate and volunteer member of the Board of Directors, Matt Plummer. Read more about his design in MDA’s Quest Media here.

“It was a great process working with MDA on this design because they allowed me to explore some different styles and really utilize my creativity in tandem with their brand in order to create something that was really special for both of us,” Plummer said. And creating a design that contributes to the progress of treatments for rare diseases is immeasurably special to Plummer. “Because of increased awareness and advocacy for rare diseases, there are more treatments than ever available to people like me,” he says. “Even just 5 to 10 years ago it seemed impossible that we would have treatment opportunities for SMA and other neuromuscular diseases. Because of MDA’s efforts and advocacy, we are now in an era where more treatments are available than ever. As a father, it’s more important to me than ever to be as healthy and strong as possible. These efforts to expand research and provide treatments help me realize that dream and realize how important every single day is.”

Social Media

MDA encourages following and sharing educational content from its national social media channels on Instagram, Facebook, X, Threads, TikTok, LinkedIn, and YouTube.

Public service announcements are available for media here.

MDA encourages everyone to join the Rare Disease Day campaign, supporting people with neuromuscular disorders and contributing to vital research and care initiatives.

About Muscular Dystrophy Association

Muscular Dystrophy Association (MDA) is the #1 voluntary health organization in the United States for people living with muscular dystrophy, ALS, and related neuromuscular diseases. For over 70 years, MDA has led the way in accelerating research, advancing care, and advocating for the support of our families. MDA's mission is to empower the people we serve to live longer, more independent lives. To learn more visit mda.org and follow MDA on Instagram, Facebook, X, Threads, TikTok, LinkedIn, and YouTube.