MDA CEO and President Dr. Wood Reflects on 2024: A Year of Milestones and Momentum

Hello MDA Families and Friends,

As we approach the close of 2024, it’s a moment for all of us at the Muscular Dystrophy Association (MDA) to reflect on the profound impact we’ve made together with our partners, supporters, and families. This year has been marked by extraordinary progress in our mission, strengthened through connection and collaboration. From groundbreaking treatments and significant legislative victories to inspiring annual initiatives like MDA Summer Camp, we have made strides in advancing care and treatment for neuromuscular diseases.

A year of major achievements

In 2024, we achieved several remarkable milestones that underscore the strength of our collective efforts. Notably:

Advocacy and legislative impact:

• One of the standout accomplishments was the inclusion of essential accessibility provisions in the FAA Reauthorization, which is a significant step in advancing the rights and access for individuals with neuromuscular diseases.
• Additionally, MDA’s Access the Vote campaign empowered voters to understand and exercise their rights during a pivotal election year, ensuring that the voices of those impacted by neuromuscular diseases were heard.

Clinical and scientific advancements:

• This year’s MDA Clinical and Scientific Conference saw an inspiring address by Brooke Eby, who shared an urgent call for continued progress in research.
• Furthermore, we saw the introduction of new treatments for myasthenia gravis and Duchenne muscular dystrophy, reflecting our ongoing commitment to advancing research and treatment.

 Community support:

• MDA celebrated the launch of its first college scholarship program, which aims to connect a new generation of advocates and supporters to the cause.

These milestones are a testament to the power of teamwork, determination, and vision. They lay the groundwork for what promises to be an extraordinary 75th anniversary year in 2025.

Investing in research and treatment

A central pillar of our mission is advancing research to find effective treatments and cures for neuromuscular diseases. This year, MDA demonstrated its continued commitment through key investments and partnerships:

• ALS research: MDA awarded a $500,000 grant to Dr. Sabrina Paganoni of Massachusetts General Hospital, supporting the HEALEY ALS Platform Trial. This pioneering initiative tests multiple therapies simultaneously to identify promising treatments for ALS, one of the most challenging neurodegenerative diseases. Dr. Paganoni highlighted the importance of collaborations like these to expedite breakthroughs that offer hope to patients and families.
• Gene therapy innovations: MDA’s Kickstart Program reached key milestones, receiving FDA designations for its gene therapy project aimed at treating congenital myasthenic syndrome caused by CHAT gene mutations. This partnership with UC Davis and Forge Biologics brings us closer to providing life-changing therapies for ultra-rare neuromuscular diseases.

Honoring advocacy and leadership

This year also saw a celebration of the outstanding advocacy and leadership within the neuromuscular community:

• MDA advocate and Major League Baseball player Rhys Hoskins was honored with the 2024 Marvin Miller Man of the Year Award and the inaugural Most Valuable Philanthropist Award by his peers. These awards recognize his leadership on and off the field and his longstanding commitment to MDA’s mission. In honor of Hoskins’ advocacy, The Players Trust awarded MDA a $50,000 grant to support our efforts.

• We are also thrilled to welcome  Barry J. Byrne to the MDA Board of Directors. A renowned pediatric cardiologist and geneticist, Dr. Byrne’s contributions to gene therapy and neuromuscular research will play a pivotal role in shaping our future strategies.

• This year, MDA introduced Ira Walker as the organization’s new adult National Ambassador, bringing fresh perspectives and insight to our advocacy efforts. Ira’s addition symbolizes the growth of our reach and the diverse voices that are now a part of our mission.

Looking ahead to 2025: 75 years of impact

As we approach MDA’s 75th anniversary in 2025, we reflect on the profound legacy we’ve built and the momentum we’ve generated in our work. The upcoming 2025 MDA Clinical & Scientific Conference in Dallas promises to be a landmark event, highlighting transformative therapies for conditions such as ALS, Duchenne muscular dystrophy, and spinal muscular atrophy. This event will bring together researchers, clinicians, patients, and advocates to share knowledge and accelerate progress.

Capitol Hill advocacy

Our grassroots advocacy continues to drive real change. In September, MDA on the Hill brought together 95 advocates for impactful meetings with lawmakers, advocating for critical bills that support the neuromuscular community. With over 97 meetings held, we worked tirelessly to promote bills such as the SSI Savings Penalty Elimination Act and the Accelerating Kids Access to Care Act, which will improve financial independence, rare disease treatment development, and access to care for children.

Gratitude and reflection

As we step into the 75th year of our mission, I offer my deepest gratitude to each of you for the focus, creativity, and dedication you bring to MDA’s work. Your commitment has made possible incredible milestones and created lasting change for families affected by neuromuscular diseases.

Before we ring in the new year, I encourage you to reflect on what this anniversary means to you, your team, and the families we serve. Together, we are stronger, and together, we will continue to make a difference.

Wishing you and your loved ones a holiday season filled with joy, peace, and a renewed sense of connection and purpose for the year ahead.

Together, we bring forward the spirit, the vision, and the commitment that define MDA.

Sincerely,

Donald S. Wood, PhD, President and CEO, Muscular Dystrophy Association