2020 is a transformational year for our community. We are excited to announce that KIF1A.ORG is one of 30 patient-led rare disease organizations chosen to join the Rare As One Project, launched by the Chan Zuckerberg Initiative (CZI) to help rare disease communities accelerate research and drive progress against rare disease. With Rare As One funding, tools, capacity-building, guidance, and collaboration, KIF1A.ORG is powered to race faster than ever to #StopTheClock and bring treatment to this generation of KAND patients.
KIF1A.ORG is a global community dedicated to improving the lives of those affected by KIF1A Associated Neurological Disorder (KAND) and accelerating research to find a cure. KAND is a rare neurodegenerative disorder caused by mutations in the KIF1A gene. KAND robs children of their ability to walk, talk, see—sometimes even eat and breathe.
KIF1A.ORG was launched in 2016 by Luke Rosen and Sally Jackson, two parents who were told there was no treatment or cure available to save their daughter Susannah from her neurodegenerative disorder. There was no clinical research initiative for KIF1A or any related disorders, so we banded together and formed a community of families, scientists, clinicians, and innovators relentlessly working toward treatment. KAND families have not only galvanized researchers to take action, in just a few years we have raised funds to support rapid and robust pre-clinical tools for therapeutic development. The Rare As One Project is a meaningful step forward in our mission to cure KIF1A Associated Neurological Disorder.
We’re proud of our progress. We’re proud of our growing community. We’re more relentless than ever. 2020 brings with it an urgent need to organize, mobilize, and drive translational research. We must scale wider and faster to improve and save the lives of this generation of KAND patients. The Rare As One Project is a collaboration that will launch KIF1A.ORG forward and empower every member of our community.
Just like our disease, KIF1A research is not as rare as we think. Our family-led organization has established strong partnerships with a core research team at Chung, JAX, and Berger Labs. But there’s more. Research teams around the world are working to understand KIF1A and similar motor proteins. We must connect and engage every KIF-related researcher to work efficiently and accelerate scientific discovery with clinically meaningful outcomes.
We don’t have the time or resources to waste on duplicative work or research that doesn’t translate from the bench to the clinic.
The Rare As One Project will power KIF1A.ORG to expand and engage a collaborative KIF1A research network working to create an efficient path to KAND treatment. Key components of our two-year project made possible by this $450,000 grant include:
- Build the KAND Research Hub: A digital space powered by KIF1A.ORG to enable KIF researchers and innovators from around the world to engage in open science through a dedicated platform for members to exchange data, challenges, ideas, and strategies to carry KAND research forward.
- Hire a Full-Time Research Coordinator: This position will primarily support our efforts to build a global, collaborative research network, manage the KAND Research Hub, and keep KAND patients/families informed and engaged in research development.
- Host a 2020 KAND Conference: KIF1A.ORG will bring together families, researchers, clinicians, and other innovators to collaborate on our strategy to accelerate the path to treatment. The 2020 KAND Conference is tentatively scheduled June 26–28, 2020, in New York City. Interested in joining us? Let us know.
The Rare As One Network will accelerate progress across the rare disease community by bringing us together to share insights and learn from one another. We thank every patient, family member, researcher, clinician, advocate and supporter for being part of the #relentless KAND community. Together, we’ll #StopTheClock on KAND.