As we reflect on the past year, we feel both humbled and inspired — humbled by the trust families place in NTSAD and inspired by the strength and resilience that define this close-knit community. 

A Year of Meaningful Progress 

NTSAD was founded by families, and 69 years later that spirit continues to guide everything we do. Our mission is to lead the worldwide fight to treat and cure Tay-Sachs, Canavan, GM1, and Sandhoff diseases by driving research, forging collaboration, and fostering community. Supporting families is the center of everything we do. It is not just what we say; it’s what we live every day. 

This year has been marked by meaningful progress and momentum. We have strengthened our support for families navigating complex diagnoses, expanded partnerships across the rare disease community, elevated the patient voice at the FDA, and continued investing in research that brings us closer to approved therapies. 

Milestones that Move Us Forward 

Two milestones this year reflect both progress and purpose. Through an extraordinary $1,000,000 gift from the founders of the Cameron and Hayden Lord Foundation, we established the Cameron and Hayden Lord Endowment, which will support ongoing research in their honor. This transformative gift not only advances science, but it also ensures that Cameron and Hayden’s legacy will continue to drive discovery and hope for years to come. 

We were also honored to receive a grant from the National Organization for Rare Disorders (NORD) to help develop our first-ever GM2 Patient Registry for all onsets of Tay-Sachs and Sandhoff diseases. This registry will play a critical role in advancing research, supporting clinical trial readiness, and ensuring that patient experiences help inform future therapies. 

“Moments like these remind us what is possible when community, generosity, and science come together,” said NTSAD CEO Kathy Flynn. “These milestones are not just organizational achievements; they are steps toward changing what’s possible for families.” 

Why This Work Still Matters 

At the same time, we recognize the reality many families continue to face. These diseases remain devastating and life-altering, and there are still no approved therapies to stop or reverse their progression. The urgency of our work has never been clearer. 

And yet — there is hope. 

Our Strategic Focus Ahead 

We are at a pivotal moment in our organization’s history. Scientific advances are accelerating, collaboration across the rare disease ecosystem is expanding, and patient voices are being integrated in drug development and regulatory decisions. NTSAD plays a unique role in bringing these efforts together while advocating for education, screening, and greater awareness. 

Looking ahead, our strategic focus reflects where we believe we can make the greatest impact. As Kathy shared, “Family support has always been the heart of our mission. As we continue to invest in research aimed at developing approved therapies that can change the trajectory of these rare diseases, we are also dedicated to prevention and to educating and empowering our community with tools and resources that enhance quality of life for individuals and families affected by Tay-Sachs, Canavan, GM1, and Sandhoff disease.” 

Expanding Advocacy and Community Voice 

We also remain committed to amplifying the voice of our community and creating more opportunities for families to engage in advocacy and awareness efforts. Every story matters, and every voice helps drive urgency and change. 

NTSAD Board President Jon Lawrence emphasizes, “Our community’s voice is one of our greatest strengths. When we come together—families, advocates, and partners—we have the power to influence decisions that will shape the future of research and care.” 

Advancing Research Toward Therapies 

We will continue advancing research and provider engagement, building on decades of investment while creating new pathways to connect clinicians and early-career professionals. The development of the GM2 Patient Registry, resources for healthcare professionals, and targeted research funding will help accelerate discoveries and improve care. 

We are also deepening strategic partnerships, as we believe that progress depends on collaboration across research, industry, and education. This includes efforts to advance carrier, prenatal, and newborn screening; support clinical trial readiness; and increase awareness of GM2, GM1, and Canavan diseases. 

At the same time, we are investing in NTSAD’s future by strengthening our ability to grow, reach more affected individuals and families, and sustaining our impact for years to come. 

Supporting Families at Every Stage 

But beyond strategy, what matters most is what this means for families. 

For those newly diagnosed, it means having a place to turn for trusted information, guidance, and connection. 

For those navigating care, it means stronger resources and a more connected network of support. 

For those waiting for treatments, it means accelerating research and removing barriers to progress. 

And for all members of our community, including those honoring loved ones, it means being part of something larger: a shared commitment to hope, connection, and change. 

No one should have to navigate a rare disease journey alone and at NTSAD, no one ever has to. 

You Are Part of this Community 

As we move forward, we want to be clear: NTSAD is not just an organization you turn to; it is a community you are a part of. As many adults and young families in our community have shared, NTSAD was never a family they expected to belong to, but it has become one they cannot imagine living without. 

Ways to Get Involved 

We invite you to get involved in whatever way feels most meaningful to you: 

• Share your story 

• Participate in advocacy initiatives such as Day of Hope or Rare Disease Day 

• Support research 

• Connect with other families 

• Engage in our educational programs and annual events 

We Are Here for You 

And if you need support, please reach out. Whether you have a question, need guidance, or simply want to connect, we are here for you and will meet you where you are. 

As Jon reminds us, “Together, we are stronger than any challenge we face. Together, we will continue to push forward towards accelerating progress, strengthening our community, and creating a brighter future for every family affected by these diseases.”