We are relentless. Today that “we” continues to grow.

Today we have a fleet of families, patients, researchers and physicians powering our urgent mission—a mission to rapidly develop treatments for KIF1A Associated Neurological Disorder. Our resilient and supportive team of partners and other committed collaborators fight relentlessly for our KIF1A community and drive us closer to the clinic every single day.

It is with science-driven hope and great thanks that we share a letter from the team at Ionis Pharmaceuticals; a team that is transforming how research is translated into potential therapies for KAND. In this time of COVID-19 disruptions, KIF1A research initiatives remain stronger than ever and research collaborators at Ionis have joined our relentless mission. Ionis Pharmaceuticals is focused on advancing their programs, including their KIF1A research program.

This letter reinforces the Ionis Team’s priority to keep their KIF1A research program on track and to maintain an open, transparent relationship with our community as they work to bring potential therapeutics to our loved ones. Ionis will continue to work with our core research team at the Chung Lab, our foundation and families to accelerate a KIF1A path to treatment.

KIF1A.ORG thanks the dedicated team at Ionis for not only leading the community in a time of crisis, but for making KIF1A Associated Neurological Disorder and our families a priority in their research programs.

Last night communities around the world stayed home and watched the Ken Burns documentary on PBS, The Gene: An Intimate History. This film is a milestone for KIF1A and many rare disease communities. We all watched as a human story unfolded; a story connecting genetics, science, medicine, biotech and families affected by rare genetic diseases. We watched Dr. Wendy Chung’s relentless commitment to scientific discovery and to all children with rare genetic diseases, including KIF1A. The Gene humanizes an epic battle that foundations like ours face daily. A battle to convince the scientific community to take action for families like ours who know time is more an enemy than nature.

KIF1A.ORG joins all of our patients and families in welcoming the Ionis Team to our army of relentless researchers. Thanks for all you do.

Click to Read the Community Statement from Ionis

With hope,

Kathryn Atchley
President, KIF1A.ORG


  1. 1

    […] trials required to build a safety profile. While we continue to work with biotech partners such as Ionis and Ovid on gene-based therapies, our new collaboration with the Christodoulou Lab is the world’s […]

  2. 2

    […] Ionis Pharmaceuticals, the world’s leader in RNA-targeted therapeutics, is in its early stages of a research program to develop a potential antisense oligonucleotide (ASO) treatment for KAND patients. Though in the early stages of this development, this is a significant impact on research of KIF1A and treatment for those living with KAND. […]

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