RAO Network

KIF1A.ORG

Luke Rosen holds his young daughter on his shoulders
KIF1A.ORG Founder Luke Rosen, his wife Sally and his daughter, who has the rare disease KIF1A. Photo by Ryan Christopher Jones.

Be nothing short of relentless

Rare disease patients and families are the bridge that connects researchers and industry, and drives them to act with urgency and an understanding of what matters most to patients. Patients and families must be at the center of R&D to deliver meaningful therapeutic outcomes. 

Luke Rosen has built a united and active community of families, scientists, clinicians, and innovators since launching KIF1A.ORG with Sally Jackson in 2016 after their daughter’s KIF1A diagnoses. KIF1A Associated Neurological Disorder (KAND) is a neurodegenerative disorder caused by gain-of-function mutations in the KIF1A gene. KAND causes a constellation of medical complexities. Symptoms first appear at birth or in early childhood. There are no approved treatments or cures, relief is symptomatic only, and it’s a degenerative disease with a progressive course.

KIF1A.ORG is excited to be part of the culture shift in the scientific and patient community that values collaboration over competition. When open science is widely embraced, KIF1A.ORG believes those at the forefront can deliver life-changing and life-saving treatment options for patients faster than ever before. The Rare As One Network will equip KIF1A.ORG with the capacity-building tools and resources needed to expand and engage a collaborative KIF1A research network so it can discover treatments for this generation of KIF1A patients.

Patients and families must be at the center of R&D to deliver meaningful therapeutic outcomes.

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