Executive Summary
In 2024, KIF1A.ORG continued its critical mission to accelerate research, foster a global community, and empower families affected by KIF1A-Associated Neurological Disorder (KAND) and KIF1A mutations. As a patient and family-led nonprofit, we bridged the gap between families, scientists, and advocates, driving patient-centered initiatives that advanced our understanding of KAND.
Key highlights include:
- Building a Global Community: Engaged a diverse set of community stakeholders across 15+ virtual events; representing patients, families, clinicians, researchers, and biotechnology leaders from around the world; grew our international ambassador representatives and offered language translation during community meetings and newly diagnosed outreach.
- Patient-Centered Research: Focused on increasing participation in our research studies, enrolling 200 patients in our Natural History Study (NHS) by year end!
- Grassroots Fundraising: Raised $250,000+ through in-person and virtual campaigns, including $70,000 for the Treatment Accelerator Program (TAP) fundraiser.
- Dedicated Staff and Volunteers: Led by Scientific Director Dr. Dylan Verden, who coordinated global KAND research initiatives and served as the central hub for all things KIF1A, our team of 10+ volunteers contributed over 3,500 hours across operations, advocacy, fundraising, research, and community engagement—driving our mission forward worldwide.
These achievements not only amplified our global presence but also solidified KIF1A.ORG as a community for hope and action in the rare disease space.
Building a Global Community
KIF1A.ORG’s commitment to fostering connection remained at the heart of our work in 2024. By prioritizing accessible, virtual programming, we expanded our global footprint, ensuring that families—from rural Australia to urban China—could access supports, resources, and participate in research.
- Virtual Community Calls: We hosted three focused community calls and recommitted to monthly meetups for newly diagnosed families. Complementing these were numerous open office hours led by our Chief Science Officer, Dr. Dylan Verden, where anyone was welcome to ask questions or discuss topics—from epilepsy strategies to daily coping tips.
- Translation services: Our meetings were enhanced by language translation software, breaking down barriers to inclusion and participation for non-English speakers. These translated sessions created a safe space for sharing experiences, celebrating milestones, and addressing challenges like ASO education and vision loss.
- Specialized Listening Sessions: In response to community feedback, we organized targeted virtual events, including an epilepsy listening session with patients and families helping to inform future patient-centered research initiatives and a specialized information session with n-Lorem discussing ASO access and treatment.
- NeuCyte Community Call and Fundraising Strategy Session: Partnering with NeuCyte, we held a call for patients and families, sparking discussions on small molecule treatments for KAND and our Treatment Accelerator Program strategy. Our Fundraising Strategy Session engaged community members interested in raising money to support our mission, and shared actionable plans that supported our families in their own fundraising strategies.
These efforts highlight our commitment to participant and topic diversity, drawing families from around the world to enhance our global presence and ensure equitable representation.
Patient-Centered Research
In 2024, our research efforts focused on expanding participation in patient-centered studies, including the Natural History Study in collaboration with Boston Children’s Hospital and the International Speech and Language Study conducted with the Murdoch Children’s Research Institute. Additionally, three pivotal scientific publications on KIF1A were published in peer-reviewed journals, underscoring the significant impact of patient participation on advancing the scientific understanding of KAND.
- Natural History Study (NHS) Enrollment: Our efforts helped achieve the successful enrollment of 200 patients in our Natural History Study (NHS) by year end. This milestone, representing a 40% increase in participation from 2023, strengthens our dataset for identifying disease symptoms and tracking potential changes over time. This work is critical to our mission and future therapeutic goals.
- Natural History Study (NHS) Publication: Dr. Wendy Chung and her team published a new Natural History Study paper in a peer-reviewed journal, providing valuable insights into KAND phenotypes, disease progression over time, and computational prediction of clinical severity. This publication represents a significant step forward in understanding the complexity of KAND—made possible through the active participation of patients and families who share their medical data, histories, and experiences. Each contribution from the KAND community directly informs the scientific process, enabling researchers to identify meaningful patterns, refine diagnostic criteria, and guide the development of potential therapies.
- First-Ever Vision Publication from KAND NHS Data: This landmark paper, co-authored by global KAND expert Dr. Wendy Chung and Dr. Aliaa Abdelhakim, detailed vision impairments in 24 NHS participants. It revealed that 95% exhibited some degree of optic nerve atrophy—a groundbreaking finding previously believed to be far less prevalent. The publication directly inspired our Vision Resource Guide, empowering families with education on KAND-specific vision symptoms and practical advocacy strategies for assessments with local practitioners, including best practices for testing and screening.
- First Antisense Oligonucleotide (ASO) Study Publication: The first KIF1A-specific ASO study was published in 2024, reporting Year One outcomes for our pioneer patient, Susannah Rosen, and highlighting safety and efficacy findings in cognition, motor function, and epilepsy. Additionally, Dr. Bain spoke on the n-Lorem podcast and gave a glimpse into Year Two and how things are shaping up for Susannah. Our second patient also started ASO treatment, Sloane and her mother Megan started a blog post so community members could follow their journey before and during treatments.
These impactful results were made possible through the dedication of both participating families and the remarkable scientists leading these studies. We extend our deepest gratitude to the researchers whose commitment, collaboration, and pursuit of discovery continue to transform patient contributions into meaningful scientific progress for the entire KAND community.
Grassroots Fundraising
Sustainable funding fuels our mission—and none of it would be possible without the dedication of our patients, families, and supporters who give their time, energy, and resources to advance this work. Grassroots fundraising continues to be a powerful driver of progress for KIF1A.ORG, and we are deeply grateful to everyone who helped sustain our mission throughout the year.
- Treatment Accelerator Program Fundraiser: Launched on KIF1A Day, April 28, 2024, the Treatment Accelerator Program (TAP) fundraiser aimed to support the next stage of research in KAND drug screening in partnership with NeuCyte. With a goal of $70,000, our community rallied together and achieved this milestone over six months—demonstrating the incredible impact of collective action.
In total, 2024 fundraising reached $250,000+, reflecting the strength and commitment of our global KAND community and the ongoing support for our mission.
Dedicated Volunteers and Staff
In 2024, KIF1A.ORG’s progress was powered by the exceptional dedication of our Scientific Director, Dr. Dylan Verden, and a team of more than ten committed volunteers. Dr. Verden served as the central hub for KIF1A research worldwide—coordinating global scientific initiatives, fostering collaborations among clinicians, researchers, and industry partners, and ensuring alignment across all facets of our research strategy. His leadership continues to strengthen our organization’s role as a unifying force in the global effort to understand and treat KAND.
Our volunteers also played an essential role, collectively contributing more than 3,500 hours across diverse areas including board service, operations, grant writing, policy and advocacy, scientific review, fundraising, conference planning, academic collaboration, and community engagement. Together, their combined expertise and passion drive KIF1A.ORG’s mission forward every day, representing more than $150,000 of in-kind support for our mission, patients, and families.
Powering Our Mission
Fiscal Management
KIF1A.ORG exists to accelerate discovery of treatments for this generation of people affected by KAND. It’s a bold mission, but not impossible thanks to our relentless community.
At KIF1A.ORG, fiscal responsibility is paramount. In 2024, we generated $294,315 in total income while incurring $148,866 in expenses, ending the year with a healthy surplus of $145,449. This prudent management—achieved with a lean team of just one full-time staff member, one volunteer Executive Director, and our extensive volunteer support—allowed us to stretch every dollar donated toward mission-critical work. Our small, dedicated core team, augmented by our volunteers, orchestrated a year of outsized impact. This efficiency model not only preserved resources for research and community programs but also built donor confidence, contributing to our 20% fundraising growth.
The following charts illustrate our income sources and expense allocations, highlighting how we directed funds strategically to fuel progress.
Looking Ahead: Planning for 2025
As KIF1A.ORG looks to 2025, we are building on the momentum of 2024 with ambitious plans that strengthen our research partnerships, expand our community programs, and drive forward therapeutic discovery.
A major highlight will be our biennial KAND Family & Scientific Engagement Conference, scheduled for July 2025 in Boston, Massachusetts. This event will once again bring together patients, families, scientists, clinicians, and industry leaders for several days of collaboration and connection. The conference will include patient and caregiver panels, research presentations, networking sessions, and opportunities for families to participate directly in ongoing studies.
In collaboration with BioSensics and Boston Children’s Hospital, KIF1A.ORG will also begin trialing digital data collection tools in our Natural History Study to better measure real-world patient experiences and motor outcomes. This initiative represents a key step toward scalable, technology-driven natural history studies that can accelerate our understanding of KAND progression and treatment response.
Our Treatment Accelerator Project (TAP) will continue advancing small molecule drug discovery through our partnership with NeuCyte, with new compound screens planned for 2025. By combining patient-driven fundraising with scientific collaboration, TAP exemplifies our mission to bridge the gap between research and real therapeutic opportunities.
Financially, KIF1A.ORG’s 2025 expense projection totals $315,686, strategically allocated to advance these priorities. The largest investments include payroll ($96,048) to sustain our core operations and leadership, NeuCyte ($70,000) and BioSensics ($50,000) to fund critical research partnerships, and the 2025 Boston Conference ($60,000) to unite our global community. Additional expenses include operating costs ($21,904), accounting ($10,930), and conference attendance ($6,804) to ensure transparency, outreach, and participation in key scientific events.
Together, these initiatives represent our next leap forward—uniting families, scientists, and innovators to turn knowledge into action and discovery into treatments for KAND.
Policy and Advocacy Highlights
In 2024, KIF1A.ORG strengthened its policy and advocacy efforts at both the state and federal levels to advance access, equity, and innovation for individuals living with rare neurologic and genetic conditions.
At the state level, KIF1A.ORG actively engaged in prior authorization reform efforts, joining a nationwide movement in which 34 states have now passed prior authorization reform to streamline access to medically necessary care. During the 2024 legislative sessions, KIF1A.ORG also submitted letters of support in Vermont, reinforcing our commitment to improving timely access to treatments for rare disease patients. In addition, KIF1A.ORG participated in the 2024 Harrisburg State Advocacy Day (September 2024) to meet directly with policymakers, share patient experiences, and advocate for policies that benefit individuals and families affected by KIF1A-related disorders.
At the federal level, KIF1A.ORG supported several key policy priorities:
- Safe Step Act – Advocated for federal protections that would require employer-sponsored health plans to provide exceptions to step therapy, ensuring patients can access the treatments their physicians prescribe without unnecessary delays.
- Rare Pediatric Priority Review Voucher Program – Raised awareness about the importance of reauthorizing this critical incentive for rare disease drug development after it was not renewed in the 2024 end-of-year package.
- National Plan for the Epilepsies – Supported efforts to establish a comprehensive national framework to reduce epilepsy-related mortality, improve diagnosis and treatment, expand research, address disparities, and increase public awareness.
- MINI Act – Backed legislation to clarify that genetically targeted therapies, including antisense oligonucleotides (ASOs), receive an appropriate exclusivity period before Medicare price negotiation, helping sustain investment in rare disease innovation.
- Home and Community-Based Services Act (HCBSA) and Home and Community-Based Services Relief Act (HCBSRA) – Supported policies to strengthen Medicaid coverage of home and community-based services, improve caregiver wages, expand respite care, and reduce waiting lists so individuals can live safely and independently in their communities.
To further elevate these priorities, KIF1A.ORG representatives also attended the 2024 Biotechnology Innovation Organization International Convention (June 2024), engaging with industry leaders, researchers, and policymakers to promote our policy positions and advocate for the urgent needs of KIF1A patients.
Together, these efforts reflect KIF1A.ORG’s ongoing commitment to shaping policies that accelerate treatment development, protect access to care, and improve quality of life for the rare disease community.
We Remember
Unfortunately, for the KAND community, progress and loss often walk hand in hand. While we celebrate every advancement and moment of hope listed above, we also grieve deeply for the superheroes taken too soon. We remember them all—the families who mourn, the children whose names we know, and the many whose stories ended before we could meet them. We invite you to pause and join us in honoring the patients and families who have endured this unimaginable loss.
KIF1A.ORG Board
As we begin a new year, we are deeply grateful for the steadfast dedication of our Board members. They continue to give their time, expertise, and energy to guide our organization with purpose—shaping strategy, strengthening fundraising efforts, and championing advocacy that moves our mission forward. Their commitment and passion are the foundation of our progress, and their leadership helps build a stronger, more connected community for all. We thank them for their continued support and belief in what we can achieve together.
Conclusion
2024 was a year of tangible progress for KIF1A.ORG. With metrics showing significant growth in our community, donations, and KIF1A-specific publications, we’re making a meaningful impact. Thank you to our community, partners, and volunteers. Together, we’re closer to reaching our mission.
For more details, visit kif1a.org or contact us at impact@kif1a.org.
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.
We hope you enjoy the following video of our warriors dancing to our favorite music: Fight Song, by Rachael Platten.