A Recap on National Muscular Dystrophy Awareness Month From the Muscular Dystrophy Association

Sharon Hesterlee, PhD

In honor of Muscular Dystrophy Awareness Month, which is celebrated every September, the Muscular Dystrophy Association (MDA) announced 70 new grants for neuromuscular disease research, totaling more than $17 million.¹ The aim of the newly funded research projects is to advance new therapies and other research discoveries in multiple areas of neuromuscular disease. Sharon Hesterlee, PhD, chief research officer, Muscular Dystrophy Association, was responsible for managing all aspects of these grant awards along with her team.

The month also kicked off MDA’s 30 Days of Strength Campaign.¹ The campaign’s purpose is to raise critical funds and awareness for MDA’s mission for empowering families all over the nation who are living with neuromuscular diseases. The events held focused on supporting and engaging those with neuromuscular diseases to have a better quality of life using the campaign slogan, “#30DaysofStrength.”¹

Hesterlee was interviewed by NeurologyLive^® about the events put on by the MDA and the importance of awareness. She mentioned the significance of hosting events for awareness of neuromuscular diseases and supporting patients in those communities. She also added that there are currently new therapies and trials in progress for neuromuscular diseases and hopes to see new technologies, such as gene therapy, be featured in future studies.

NeurologyLive^®: What events took place for Muscular Dystrophy Awareness month?

Sharon Hesterlee, PhD: During Muscular Dystrophy Awareness Month, we held our MDA Tribute Tour–St. Louis. During the tour, we awarded MDA Tribute Awards to Dr. Alan Pestronk of Washington University School of Medicine; Mike Bush, legendary anchor of NBC's KSDK-TV 5 On Your Side; and the MDA Muscle Walk team 'The Flying Pigs', comprised of the Hawn and Bliss Holler families. The awards were part of the 35th Annual 'Show of Strength' hosted by Bush on Sunday, September 4, broadcasting in primetime on KSDK-TV live from Grants Farm in St. Louis, Missouri.

At the end of the month, MDA held our Virtual Summit and Hill Day. MDA advocates from across the country met virtually with lawmakers to make their voices heard and urged elected officials to take action on MDA’s legislative priorities, including access to care, accelerating therapies, and empowerment and independence for people with disabilities.

Our educational programming during Muscular Dystrophy Awareness Month included educational events such as our Duchenne Muscular Dystrophy Symposium, and our engage webinar on genetic testing for Becker muscular dystrophy. Finally, MDA Let’s Play is a community united by the love of gaming and support for MDA’s mission. The community plays weekly game nights on Saturdays at 7:00 PM ET. On World Duchenne Day, September 7, streamer Charlie Harley, who lives with Duchenne muscular dystrophy, hosted a fundraiser for MDA.

What events would you like to see in the future for raising awareness on muscular dystrophy?

We hope people stay engaged with us all year long, follow our work and progress on social media @MDAorg, and engage in our 2023 MDA Clinical & Scientific Conference when we convene the global neuromuscular community in-person and virtually.

Why do you think it is important to host these events for awareness?

Disease months such as Muscular Dystrophy Awareness Month are important times to recognize our longstanding legacy with valued supporters, clinicians, researchers, and many others providing this pipeline of progress that is the hallmark of today's neuromuscular disease frontier.

What are your thoughts on newborn screening for muscular dystrophy?

MDA Advocacy work aims to expand and improve the newborn screening program, which is vital to ensuring all babies are screened shortly after birth for neuromuscular diseases. Earlier this summer, MDA officially cosponsored the nomination for Duchenne muscular dystrophy (DMD) to be added to the Recommended Uniform Screening Panel (RUSP) for newborn screening. With the advent of life saving treatments (ie, in the example of SMA), some funded from MDA research, newborn screening is particularly important. For additional information, watch “MDA Advocacy Institute: The Future of Newborn Screening.”

What new research studies are being conducted on muscular dystrophy?

There are more than 100 active studies of new therapies for the muscular dystrophies underway. In particular, there are 3 gene therapy studies in progress for DMD and several additional studies testing small molecules or “biologics”—drugs based on protein or DNA—for DMD and Becker muscular dystrophy. There are also trials in progress for limb-girdle muscular dystrophy, myotonic muscular dystrophy and facioscapulohumeral muscular dystrophy. In the future we expect to see studies of new technologies like gene editing and nonviral gene delivery.

What are your reflections on Muscular Dystrophy Awareness Month, and is there any news happening with MDA?

We are grateful for how our community and the public engaged in Muscular Dystrophy Awareness month and we continue our work through fundraising partnerships to support our mission to fund our MDA Care Center Network—the research that we fund globally and share freely—and our advocacy and educational programming. We also always want to amplify that MDA is here for the community through our MDA Resource Center for guidance and support when someone is diagnosed with neuromuscular disease.

Coming up on October 27, we’re excited to bring the MDA Tribute Tour to Boston, for the MDA Tribute Awards honoring Louis Kunkel, PhD, of Boston Children’s Hospital; Dr. Brenda Wong, MD, of UMass Chan Medical School; Natalie Jacobson, former anchor WCVB-TV; and Tom Graney, founding family of IAFF Fill the Boot. The MDA Tribute Awards in Boston are notable because they honor individuals who have made extremely significant contributions to progress for people living with DMD through scientific discovery, care, and fundraising. MDA has a strong legacy of innovation around DMD, particularly in Boston, and these awards honor that heritage.

We are also always sharing stories from our community on our Quest Media platforms including the Quest blog, podcast, magazine, newsletter. This month in October, during National Disability Employment Awareness Month, we have new MDA Access with “Access to Employment” programming, and Diversity Equity, and Inclusion Coalition content all aimed at disability disruption and amplification of the equity equation.

REFERENCES
1. Muscular Dystrophy Association. Muscular Dystrophy Association Kicks Off 30 Days of Strength Campaign for National Muscular Dystrophy Awareness Month in September. PR Newswire. August 22, 2022. Accessed October 18, 2022. https://www.prnewswire.com/news-releases/muscular-dystrophy-association-kicks-off-30-days-of-strength-campaign-for-national-muscular-dystrophy-awareness-month-in-september-301610022.html