From KIF1A.ORG April 24, 2026April 24, 2026

Executive Summary

In 2025, KIF1A.ORG continued its critical mission to accelerate research, strengthen a global community, and empower individuals and families affected by KIF1A-Associated Neurological Disorder (KAND) and KIF1A mutations. As a patient- and family-led nonprofit, we bridge the gap between families, scientists, clinicians, and advocates—driving patient-centered initiatives that advanced research, improved care infrastructure, and deepened community connection. 

2025 was a year of meaningful progress and important growth. We welcomed new leadership, expanded our Board of Directors, and hosted the 2025 KAND Family & Scientific Engagement Conference in Boston. We launched our first KIF1A Virtual Hill Days, funded two new patient-centered research studies, and celebrated the first formal KIF1A Center of Excellence at Boston Children’s Hospital. On top of it all, we had one of the strongest financial years in KIF1A.ORG history – raising $1,865,094 to advance our mission. These milestones reflect the strength of a community that continues to show up with urgency, resilience, and hope. 

Key highlights include:

• Leadership & Organizational Growth: Welcomed Dominique Lessard, Ph.D., as Executive Director and expanded board leadership with the addition of Sarah Goldberg during a pivotal period of transition and growth.
• Global Community Growth & Connection: Welcomed 109 new patients and 4 new International Ambassadors to our global community in 2025 and facilitated meaningful opportunities for learning, collaboration, and belonging through virtual events and the 2025 KAND Family & Scientific Engagement Conference.
• Policy & Advocacy in Action: Mobilized the KIF1A community around Rare Disease Day through our first-ever Miles that Matter for KIF1A Virtual Challenge and launched our first-ever KIF1A Virtual Hill Days – bringing patient voices directly into legislative conversations around Medicaid protection, the MINI Act, and the Accelerating Kids’ Access to Care Act.
• Patient-Centered Research: Expanded KIF1A patient-centered research through two important pilot initiatives: an 8-week, two-patient trial using SpineX’s SCiP neuromodulation device, and a partnership with BioSensics to explore wearable digital data collection as part of the KOALA Study.
• Clinical Care & Infrastructure: Helped lay the groundwork for the first-ever formal KIF1A Center of Excellence at Boston Children’s Hospital, a landmark step forward in KIF1A-specialized clinical care. 
• Powering the Mission: Raised $1,865,094 in 2025 through a combination of grassroots fundraising, sponsorship, general donations, investment return, and transformational philanthropic support—creating one of the strongest financial years in KIF1A.ORG history.

2025 was not just a year of activity – it was a year of building. We strengthened research infrastructure, expanded organizational leadership, invested in community programs, elevated our policy voice, and helped inform emerging models for better clinical care. Together, these efforts are helping move the KAND community closer to treatments, better support, and a stronger future.

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Leadership & Organizational Growth

During this pivotal year, KIF1A.ORG also expanded and strengthened its Board of Directors. As Dominique transitioned into the Executive Director role, she joined the Board of Directors, officially filling Angie Fuller’s former board seat. At the same time, Angie was elected to the Board, ensuring continuity of leadership while carrying forward the invaluable perspective of a longtime advocate, parent leader, and former Executive Director.

KIF1A.ORG was also proud to welcome Sarah Goldberg to the Board of Directors. Sarah brings valuable experience in legal counsel, business strategy, data management, nonprofit leadership, and organizational growth, adding important perspectives as KIF1A.ORG continues to expand its reach and deepen its impact. Her appointment reflects the organization’s ongoing focus on broadening expertise, increasing capacity, and building a strong, mission-aligned board prepared to guide KIF1A.ORG through its next chapter of growth.

As the year progressed, KIF1A.ORG continued to strengthen its operational capacity. In November, the organization announced plans to hire a part-time Director of Development to support fundraising strategy, donor relations, and community engagement – an important step in growing the organization’s development infrastructure. During the fourth quarter community call, the team also shared progress on the development of a five-year strategic plan, underscoring a clear focus on sustainability, long-term growth, and mission-driven impact. 

This year’s progress was made possible by the dedication of staff, board members, volunteers, community leaders, and family advocates who continue to move this mission forward with passion and purpose. As in years past, KIF1A.ORG’s ability to do more with limited resources remains one of its greatest strengths.

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Global Community Growth & Connection

At the heart of KIF1A.ORG’s work is the belief that no family should face KAND alone. In 2025, we welcomed 109 new patients and families to our growing global community, further expanding the reach and strength of the KAND network. We also continued to build a stronger, more connected global community through shared learning, meaningful engagement, and opportunities for families, clinicians, researchers, and advocates to come together. Whether through quarterly community calls, newly diagnosed outreach, educational resources, or planning sessions tied to research and conference participation, KIF1A.ORG remained a trusted home base for families navigating life with KAND. 

A key part of that global connection is our International Ambassador Program. In 2025, we welcomed 4 new ambassadors, bringing our total to 18 ambassadors representing 13 countries and regions around the world. Our ambassadors play a vital role in connecting families across languages, cultures, and time zones—helping strengthen relationships, expand access to information, and ensure our global community feels seen, supported, and included.

A defining highlight of the year was the 2025 KAND Family & Scientific Engagement Conference, held July 25–26 in Boston, Massachusetts. This event brought together 320+ patients, families, researchers, clinicians, and supporters from across the globe for one of KIF1A.ORG’s most important opportunities for connection and progress. With both in-person and virtual options, the conference was designed to maximize accessibility while creating space for research, scientific exchange, shared learning, and community-building. 

In August, KIF1A.ORG extended the reach of the conference by sharing session recordings, downloadable slides, and a highlight video so families who could not attend could still benefit from the experience. To further support accessibility and inclusion, the conference also offered real-time language translation in more than 50 languages and used the polling platform Slido to encourage real-time participant feedback, audience engagement, and live Q&A. These efforts reflect KIF1A.ORG’s ongoing commitment to making every major community moment as accessible, inclusive, and participatory as possible. 

KIF1A.ORG also continued to prioritize connection across every stage of the journey. In December, the organization invited newly diagnosed adults with KIF1A mutations to express interest in future virtual meetups, continuing to expand support pathways for often-overlooked parts of the community. Across every format – virtual, in-person, scientific, emotional, and practical – KIF1A.ORG continued to create spaces where families could find information, support, and belonging. 

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Policy & Advocacy in Action

In 2025, KIF1A.ORG continued to elevate patient and caregiver voices in policy spaces, recognizing that scientific progress alone is not enough without access, coverage, and systems that support rare disease families. Throughout the year, the organization expanded its advocacy efforts to ensure the needs and lived experiences of the KAND community were represented in broader healthcare and legislative conversations.

In February, KIF1A.ORG joined the global rare disease community in honoring Rare Disease Day, using the month as a powerful opportunity to build awareness, elevate patient stories, and inspire action. As part of this effort, the organization launched its first-ever Miles that Matter for KIF1A virtual challenge, inviting participants to walk, roll, or move 28 miles over the 28 days of February. The response was incredible: over 850 participants joined the challenge, raising awareness of KAND with every mile walked, story shared, and conversation sparked across the rare disease community. Altogether, we logged 25,000 miles – enough to circle the Earth once!

In June, KIF1A.ORG amplified a national call to action urging supporters to speak out against proposed Medicaid cuts that could create major barriers to care for rare disease patients. By sharing advocacy opportunities and encouraging families to contact their Senators, the organization reinforced its commitment to protecting access to the healthcare systems that many KAND families rely on every day.

One of the most significant advocacy milestones of the year was the launch of KIF1A.ORG’s inaugural Virtual Hill Days, sponsored by Novartis. First introduced in September and held successfully in October, this new initiative connected KIF1A families and advocates with their local legislative offices across the United States. Participants met with members of Congress and their staff to advocate for key policy priorities, including the MINI Act and the Accelerating Kids’ Access to Care Act.

KIF1A.ORG defined three clear goals for this effort:

• Connect KIF1A families and advocates with their legislative offices to amplify their voices
• Advocate for policy priorities that matter to the KAND community
• Bring legislators into the lived reality of KAND through storytelling and shared perspective

This was a major step forward for the organization. In 2025, KIF1A.ORG was not only responding to policy conversations – it was building the infrastructure, confidence, and community voice needed to help shape them.

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Patient-Centered Research

In 2025, KIF1A.ORG remained steadfast in its commitment to patient-centered research by helping move the community from enrollment to meaningful participation. After reaching the major 2024 milestone of 200 patients enrolled in the ASCEND online natural history study, the organization focused on what comes next: completing surveys, re-engaging participants, strengthening longitudinal follow-up, and expanding complementary research efforts that deepen our understanding of KAND. 

In February, KIF1A.ORG launched the ASCEND Survey Completion Challenge, emphasizing that enrollment is only the beginning. The more complete and consistent the data, the more powerful the study becomes for researchers working to understand disease progression and prepare for future therapeutic development. In partnership with the Chung Lab, KIF1A,ORG encouraged participants to complete their initial assessment and annual updates, reflecting the organization’s shift from recruitment to completion and underscoring a research strategy focused not only on participation numbers, but on data quality and long-term scientific value. By the end of 2025, 352 patients were enrolled in the ASCEND study.

Throughout the spring and summer, KIF1A.ORG actively supported participation in the KOALA Study, especially in conjunction with travel to Boston for the 2025 conference. Families were encouraged to combine research visits with conference attendance to reduce logistical burden while maximizing scientific impact. In May, the organization explicitly noted that KIF1A Day fundraising would help facilitate patient travel for KOALA study visits during the summer in Boston – an important example of how donor support directly reduces barriers to research participation. By the end of 2025, 97 patients had enrolled in KOALA study visits. Below are images of one of our patients participating in several of the assessments associated with the study.

In late summer and fall, KIF1A.ORG and Dr. Wendy Chung’s team made a coordinated push for medical record collection as part of the ASCEND study. Families were asked to submit medical records to help fill critical gaps left by surveys and assessments alone. This work is essential to creating a more complete clinical picture of KAND over time and reflects a more sophisticated, longitudinal approach to natural history research. 

By the end of 2025, these studies included patients representing 40 countries—underscoring both the global reach of this work and the diversity of the community it serves. This level of international participation reflects a broad range of genetic backgrounds, healthcare systems, and lived experiences, strengthening the quality and relevance of the data. It also highlights the growing awareness of KAND worldwide and the collective commitment of families, clinicians, and researchers across borders to advance understanding and accelerate progress toward treatments.

Exploring New Therapeutic Pathways

In addition to natural history and clinical data collection, 2025 also reflected KIF1A.ORG’s continued commitment to exploring broader opportunities that may improve quality of life and future care. Throughout the year, the organization remained focused on supporting collaborations and building the scientific ecosystem needed to move from observation to intervention.

One key initiative was an 8-week, two-patient pilot study using SpineX’s SCiP neuromodulation device. Coordinated with a specialized physical therapist in Southern California and led in partnership with SpineX founder Parag Gad, this study tested patient mobility before, during, and after 8 weeks of physical therapy sessions while using this device. The SCiP device, applied via skin patches during physical therapy, modulates nerve signals along the spine – similar to how a hearing device adjusts sound for someone who is hard of hearing – helping nerve signals reach their intended target more effectively. One patient showed improved independence and balance when walking, while the other did not see significant mobility changes. Next steps include preparing a publication on the pilot study and determining whether to expand the study to additional patients. In 2025, the SCiP device was under review for FDA approval but had not yet received clearance.

In parallel, KIF1A.ORG partnered with BioSensics to pilot wearable digital data collection for the KOALA Study. Launched in conjunction with the 2025 KAND Family & Scientific Engagement Conference, more than 40 patients took home a set of non-invasive devices – including a smartwatch, pendant, and ankle monitors – to be worn for one week and then returned. These devices capture around-the-clock data on temperature, pulse, sleep patterns, gait, balance, activity level, and more, providing a real-world view of patient function. Once returned and analyzed, the data will help determine whether wearable digital data collection should become a long-term component of the KOALA Study – offering a promising way to include patients who are unable to travel to Boston due to complex medical needs, expand access to research participation, and capture valuable real-world data that could significantly deepen our understanding of KAND.

Every survey completed, every medical record shared, every research visit attended, and every family that says “yes” to participation helps move the field forward. In rare diseases, progress depends on partnership – and KIF1A.ORG continues to make that partnership possible between researchers and patients.

We extend a heartfelt thank you to all of our research participants for their generosity, courage, and commitment—your contributions make every advance possible.

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Clinical Care & Infrastructure

One of the most significant milestones of 2025 was the announcement that Boston Children’s Hospital will launch a KIF1A NEXTGen Clinic in 2026 – the first-ever formal KIF1A Center of Excellence for this community. This is a landmark step forward in KIF1A-specific clinical care and a powerful reflection of years of patient-led advocacy, research participation, and partnership-building with Dr. Wendy Chung and Boston Children’s Hospital leadership. 

The KIF1A NEXTGen Clinic will bring together a specialized, multidisciplinary group of providers within Boston Children’s Hospital to deliver evidence-based, patient-centered care tailored specifically to the needs of individuals with KIF1A mutations. This is not simply about access to appointments – it is about access to informed and coordinated expertise. 

In December, KIF1A.ORG launched the 2026 Community Clinician Survey to help shape the next chapter of care. Families were asked to share information about the specialists they currently see so the organization could support the continued development of the KIF1A NEXTGen Clinic, build a database of experienced clinicians and their locations, identify potential experts, and help newly diagnosed families connect with knowledgeable providers closer to home. This work reflects KIF1A.ORG’s growing role not just in research acceleration, but in clinical systems design – helping define what better care can and should look like for rare disease families.

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Powering Our Mission

In 2025, KIF1A.ORG’s fundraising and financial stewardship created one of the strongest years in the organization’s history – powering critical progress across research, advocacy, family support, and long-term organizational growth.

Thanks to the extraordinary generosity of families, donors, volunteers, sponsors, and community champions around the world, KIF1A.ORG raised $1,865,094 in total revenue. This remarkable year was fueled by a combination of grassroots fundraising, sponsorships, year-round giving, investment return, and transformational philanthropic support, including a landmark $1.6 million gift from Las Condes Trust. At the same time, community-driven fundraising remained a vital force, with Miles that Matter for KIF1A raising $67,432, general donations contributing $173,339, and corporate sponsorships adding $15,000.

These funds did more than sustain operations – they directly accelerated mission delivery. In 2025, donor support helped reduce barriers to research participation, including travel support for KOALA study visits in Boston; advanced patient-centered research efforts such as ASCEND, KOALA, the SpineX SCiP pilot, and BioSensics wearable data collection; strengthened advocacy through Rare Disease Day and the launch of KIF1A.ORG’s first-ever Virtual Hill Days; and supported family-centered programming, including the 2025 KAND Family & Scientific Engagement Conference. It also helped expand the staffing, systems, and infrastructure needed to meet the growing needs of the KAND community and prepare for the future.

KIF1A.ORG spent $453,543 in 2025 to support mission-critical work, with investments focused primarily on payroll ($209,700), research and development ($101,600), and the 2025 conference ($84,776) – reflecting a clear commitment to building both the scientific and organizational capacity required to drive progress. Direct assistance to families, conference attendance support, and essential operating costs further underscored the organization’s focus on thoughtful, transparent, and mission-aligned stewardship.

Looking ahead, KIF1A.ORG enters 2026 with strong momentum and a strategic plan for continued growth, including a projected $688,385 in mission investment – anchored by increased spending in research and organizational capacity. This reflects a deliberate commitment to funding the people, partnerships, and infrastructure needed to accelerate progress for individuals and families living with KAND.

Every dollar raised in 2025 helped build a stronger foundation for the future. It powered programs, expanded possibility, and moved the KIF1A community closer to more answers, better care, and greater hope.

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We Remember

For the KAND community, progress and heartbreak walk hand in hand. KIF1A Associated Neurological Disorder (KAND) is a devastating neurodegenerative condition that robs children of abilities, shortens lives, and places immense emotional and physical burdens on families. This reality of loss and struggle sits alongside every accomplishment, reminding us why our mission matters so urgently. Every research breakthrough, every family supported, every policy meeting, and every community milestone is held alongside the memory of the superheroes we have lost. 

In September, KIF1A.ORG honored the life of Livia Bottini Elizardo (December 3, 2023 – August 12, 2025), a cherished KAND warrior from Brazil whose family shared the deep love, joy, and heartbreak of her journey. In their words, Livia brought light, taught unconditional love, and transformed their lives in the short time she was with them – leaving a legacy that resonates with all who knew her.

We remember Livia and all those taken too soon, and we carry their courage with us in every step forward.

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Looking Ahead to 2026

As KIF1A.ORG looks to 2026, we do so with strong momentum, a stronger infrastructure, and a clearer path forward.

Key priorities already underway include:

• Launching the first-ever KIF1A Center of Excellence at Boston Children’s Hospital 
• Using the 2026 KIF1A Community Clinician Survey to shape more coordinated, KIF1A-informed care pathways 
• Continuing to grow participation in research studies and deepen the quality of longitudinal clinical data
• Building on the success of Virtual Hill Days to strengthen KIF1A’s policy voice in healthcare and research conversations 
• Investing in organizational growth through fundraising infrastructure and strategic planning 
• Preparing for another year of community connection, awareness, and action through 2026 events and campaigns, including Miles that Matter, KIF1A Week, and Giving Tuesday

The work ahead is ambitious – but so is this community.

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Conclusion

2025 was a year of meaningful progress, organizational growth, and foundational work for the future.

KIF1A.ORG strengthened research participation, supported families in accessing studies, brought the community together in Boston for a powerful and unifying conference, launched its first-ever Hill Days, expanded leadership and governance, and helped catalyze the development of the first formal KIF1A Center of Excellence. These are not small wins – they are the building blocks of a stronger future for every family affected by KAND. 

To every patient, caregiver, volunteer, donor, researcher, clinician, advocate, sponsor, and partner: thank you.

Your courage, generosity, and belief in this mission continue to make life-changing and life-saving progress possible.

From all of us at the Board of Directors and the Executive Director’s office: together, we are moving research forward, improving care, amplifying voices, and building a future with more answers and more hope for every KAND warrior.

KIF1A.ORG Board

No matter what your role is in our community, we thank you for joining our relentless mission. You’re making life-changing and life-saving impacts on countless superheroes today and in the future.