Stepping into this role in such a historic year has been humbling and inspiring. This is my first End of Year message as MDA’s President and CEO, and I want to begin by thanking you. Our progress in 2025 was driven by the partnership we share across this entire community of families, clinicians, researchers, volunteers, advocates, donors, and the MDA Ambassadors who bring this mission to life transparently and with courage.
Gratitude for MDA Ambassadors and the Community Advisory Task Force
Our MDA Ambassadors, led by National Ambassadors Lily Sander and Ira Walker, touched every corner of this mission in 2025. Their advocacy influenced policy conversations, fundraising campaigns, media coverage, and community events. Their willingness to speak from lived experience helps the world understand neuromuscular disease with greater honesty and depth.
Members of our Community Advisory Task Force guided us with candor. Their insights support decisions about MDA research priorities, educational programs, care delivery, direct services, and accessibility. Their leadership continues to shape what MDA becomes over the next 75 years.
A historic year for science, treatments, and momentum
The 2025 MDA Clinical and Scientific Conference once again convened the largest gathering of global neuromuscular experts, researchers, advocates, industry partners, and families. The conference highlighted new science, evolving standards of care, real world data, and early insights into emerging therapies. It also created space for essential conversations about patient experience, clinical trial access, equity in care, and the responsible development of advanced technologies. The energy and collaboration across sessions reflected a field that continues to move quickly and thoughtfully. MDA’s annual conference is still one of the most important platforms for shaping the future of neuromuscular research and care, and I am grateful to every speaker, attendee, and partner who made this year’s gathering such a powerful catalyst for progress in neuromuscular disease research and care.
Our 75th anniversary year delivered milestones that speak to decades of discovery and scientific rigor. Multiple FDA approvals expanded treatment options across neuromuscular diseases.
- Thanks to research funding provided by MDA, KYGEVVI is now available as the first therapy for thymidine kinase 2 deficiency, one of the rarest mitochondrial disorders.
- ITVISMA became the first gene replacement therapy available to older children, teens, and adults living with spinal muscular atrophy.
- IMAAVY advanced treatment for people living with generalized myasthenia gravis.
These breakthroughs reflect the power of scientific persistence and the long arc of investment in early career researchers, many of whom received MDA funding at pivotal stages in their work. In 2025, we awarded and co-funded five million dollars in research grants to propel the next generation of discovery, with more grants coming in early 2026.
This year also reminded us that scientific progress carries real human cost, and that progress is never separate from grief. Some deaths were the result of disease progression. Others occurred during participation in gene therapy research, where people and families made the extraordinary decision to step forward in the hope of advancing knowledge and expanding options for those who would come next. Their courage matters.
Gene therapy remains one of the most promising paths forward for many neuromuscular diseases, but its future depends on careful study, transparent reporting, and shared accountability among researchers, clinicians, regulators, and families. MDA is committed to advancing this work both urgently and responsibly, centered on safety, equity, and the wellbeing of the people who place their trust in emerging treatments. We carry forward the memory of those we lost by insisting that progress be worthy of their courage.
Earlier Answers for Families: Duchenne Added to the RUSP
The addition of Duchenne muscular dystrophy last week to the Recommended Uniform Screening Panel (RUSP) is a powerful moment of progress for the Duchenne community and a transformative step forward for newborn screening nationwide. It means that more families will receive answers sooner, at the very beginning of their journey, when knowledge can bring direction, confidence, and possibility.
This milestone reflects years of determined advocacy, leadership, and belief in what early diagnosis can change. It was driven by families who shared their stories, advocates who refused to let this moment pass, and clinicians and researchers who move science into action. Together, they helped ensure that Duchenne is recognized early, giving families the time and information they deserve to plan, to act, and to hope.
MDA is proud to work alongside this community as the recommendation moves toward implementation in every state in the nation.
High-Impact Engagement, Delivered Locally
The MDA Engage events in Dallas and San Francisco this fall delivered unique learning and networking opportunities for people living with neuromuscular disease and their caregivers. Both symposiums drew in-person participation from families eager for practical, expert-led education, peer connection, and trusted resources, reinforcing the value of no-cost, community-centered programming that meets people where they are and supports real-world care and life decisions.
Advocacy at a time when families cannot afford to lose ground
Our largest Hill Day in MDA history made clear that the voice of this community carries real weight.125 advocates, ranging in age from seven to 70 met with lawmakers to defend Medicaid access, strengthen NIH funding, improve home care policies, expand newborn screening coverage, and protect the programs that allow people with neuromuscular disease to live with strength and independence.
This rallying point came at a critical moment. Federal agencies that drive rare disease research face cuts, and many essential disability supports remain under pressure. Your advocacy made the stakes visible and personal, and we will continue to grow this national presence in 2026.
Care Centers, camp, and direct support for families
Expert care is still central to our mission. MDA Care Centers and Affiliates continued to deliver multidisciplinary guidance, clinical trial access, and coordinated support that families rely on in over 150 locations. Our work with clinicians this year focused on strengthening quality, consistency, and readiness for emerging therapies that require careful long-term monitoring.
Summer Camp brought nearly 900 children experiences that help shape confidence and resilience well into adulthood. Campers tried new activities, formed friendships, and connected with volunteers who created safe and joyful environments. These weeks represent the spirit of MDA at its best.
Beyond these signature programs, donor generosity made it possible to expand grants for durable medical equipment. For many of our families, insurance will not cover the full cost of equipment that greatly improves quality of life and participation for people in our community. Often, the funding gap is too much for the family to cover without financial help. This support is a vital part of how we meet families where they are.
A new model of sustainable funding
This year also marked the establishment of the Helen Paves Fund for Care and a Cure, an endowed fund created with beauty expert and philanthropist Ken Paves in loving memory of his mother, Helen. We expect the fund will grow to at least $5 million over the coming year and will support ALS care through what is now known as the Helen Paves MDA ALS Care Center Network. Helen lived her life helping other ALS families find compassionate, gold standard care, and this fund continues that legacy.
Honoring the service of Dr. Don Wood, our board, our management team and staff members, and MDA volunteers
This year included a transition. Our former President and CEO, Dr. Don Wood, concluded his tenure with MDA after many years of dedicated service as CEO, as a member of the Board of Trustees, and as a researcher, including his early work as an MDA grantee. Dr. Wood cared deeply about strengthening MDA’s support for families, advancing research partnerships, and elevating the importance of community connection. We thank him for his leadership and wish him well in his next chapter.
Our mission and fundraising volunteers gave their time, energy, and lived experience to support families, strengthen programs, and extend MDA’s reach in communities across the country. So did our all-volunteer Board of Directors, who provide MDA with steady guidance, thoughtful oversight and a clear commitment to the long-term strength of the mission and the MDA brand.
Our donors make MDA’s work possible. Their trust, generosity, and belief in MDA’s mission sustained families, accelerated research, and strengthened care and community programs during the year. Their commitment continues to power both the progress we made in 2025 and the momentum carrying us forward into 2026 – and beyond.
I also want to call our legacy partners, including CITGO and the International Association of Fire Fighters, who have supported MDA for decades. The enthusiasm we receive from every stratum of these organizations – from employees bidding with good-natured competitive energy on auction items to support MDA, to fire fighters who stand on street corners from coast to coast to Fill the Boot – we are deeply grateful for your partnership and trust in our ability to advance our mission.
Meanwhile, MDA staff brought dedication and skill to every corner of MDA. They supported our families, advanced research, strengthened partnerships, delivered national campaigns to reach and engage our community, and upheld the values that define this organization. I am deeply grateful for their service.
Closing a milestone year and stepping into the future with you
Ending our 75th anniversary year carries pride and reflection in equal measure. We honored a powerful legacy. We saw breakthroughs once thought out of reach. We saw young people thrive at camp, and families receive expert guidance at Care Centers. We stood together on Capitol Hill and made the voices of this community impossible to ignore.
We also recognize what is still ahead of us. Families still face barriers to diagnoses and care. Scientific progress depends on consistent, protected research funding. Equity in access, education, and support requires constant attention. These realities shape our path and commitment for the years ahead.
The next era of MDA will deepen our work across research, care, advocacy, and community building. We will strengthen programs for people at every stage of life. We will continue to invest in emerging therapies and the clinical infrastructure that surrounds them. We will expand support services and education for families. And we will bring this mission to more people nationwide.
Above all, we will move forward together. This community is a partner in every success, every breakthrough, and every lesson learned. Thank you for your trust, your insight, your energy, and your belief in what we can build.
I look forward to our next year of progress and to the future we will shape together.
SH
PS. Would you like to engage with us in 2025?
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