KIF1A.ORG was established in 2017 as a parent-led organization dedicated to discovering a cure for children living with KIF1A associated neurological disorder (KAND). KAND causes a group of related severe medical conditions that include spastic paraplegia, cerebellar atrophy, epilepsy, cognitive impairment, optic nerve atrophy, and peripheral neuropathy. These conditions are progressive and are associated with neurodegeneration, igniting a fierce sense of urgency in the project team’s mission.
With approximately 200 known cases of KAND, the community is small, with limited numbers of identified patients, and challenged by limited disease understanding. The team sees this challenge as an opportunity to establish a culture of patient-centered research and development of a strategic research plan that is focused on improving quality of life, enhancing clinical care, and developing treatments. Through the KAND Family and Scientific Engagement Conference, the project team aims to educate the community of KAND families and empower them to engage as partners in KAND research and help establish the priorities and research agenda.
Working closely with Wendy Chung, MD, PhD, from Chung Lab at Columbia University Medical Center, KIF1A.ORG volunteers will plan and execute the conference in August 2019 at Columbia University in New York. The two-day event will drive transparent and meaningful dialogue between KAND caregivers and researchers. Sessions will address topics such as an introduction to patient-centered outcomes research, KAND research updates and strategy, prioritization of treatment, and how caregivers can drive KAND research and develop a strategic plan.
The conference will be the largest gathering of KAND caregivers and researchers to date. The team aims for each participant to leave the conference invested in establishing and nurturing a patient-centered approach to KAND research. The team also aims to help caregivers improve the health and lives of KAND patients immediately while laying the foundation for long-term treatments.
The conference will include interactive sessions related to KAND research and collaboration on a patient-centered road map toward a cure. Activities include Q&A sessions, roundtable discussions, and group brainstorming to foster open conversation.
- Creation of patient-centered KIF1A.ORG action plan
- Copies of session materials available to the public
- Number of caregiver participants: 100
- Number of researcher participants: 25
- Number of caregivers interested in joining a new KAND Caregiver Advisory Board to prioritize and cultivate a patient-centered culture
- Percentage of caregivers with increased knowledge of KAND research
- Percentage of caregivers receiving new information that will enable them to make better healthcare decisions (e.g., try new therapy or medication)
- Percentage of researchers who have an increased understanding of patient/caregiver perspectives, which will drive the development of meaningful patient outcomes
A conference planning committee will include KAND parents and researchers. The committee will meet monthly beginning fall 2018 and more frequently leading up to the conference. Postconference follow-up and the creation of a KAND Caregiver Advisory Board will ensure ongoing patient-centered engagement.
KIF1A.ORG is proud to partner with Chung Lab, Columbia University Medical Center, Albert Einstein Medical College, and The Jackson Laboratory to make this collaborative conference possible.