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Hello Friends

We are gearing up for major success this year that is both mission-focused and community-driven. Our pathway to treatment is the compass leading the way. Join us each month as we spotlight our monthly momentum and impact!

Research Highlights

Newly Funded Project: Rarebase

Our pipeline of therapeutic development projects is growing! KIF1A.ORG is partnering with Rarebase to explore a potential therapeutic avenue for KAND using antisense oligonucleotide (ASO) technology. Rarebase is a public benefit corporation pioneering a new approach to biotech by partnering with patient organizations to identify new therapeutic options with the highest probability of success. Read more about this new partnership and other projects made possible by the KIF1A.ORG community of families, researchers and supporters like you!

Community Highlights

Save the Date for Upcoming Events


KIF1A.ORG’s 2021 Virtual Conference: July 9-10, 2021

Our community conference will be hosted virtually this year on July 9th and 10th. Great topics and opportunities to connect with caregivers, patients and the scientific community are in the pipeline for this event! We can’t wait to share more information with all of you in the very near future.

KIF1A Day Is April 28th!

Our KIF1A community can help translate awareness into action on this day by encouraging family and friends to support our efforts. Be on the lookout for our special KIF1A Day plans!

KAND Clinician Directory Development

As another resource for patients and caregivers, we wish to connect our community with the most talented and enthusiastic clinicians in the world who support our KAND community. We are establishing a global directory of clinicians, who are highly regarded in our community, as an available resource when new families join our community or for those in need of recommended options within their geographical location. Families can complete the KAND clinician survey to help build our directory.

Looking Back at Rare Disease Day Success!

Our Rare Disease Day fundraising efforts through Facebook and our website raised a total of $22,874.08 between February 22nd and March 4th!

Our community has always been fierce with their fundraising efforts, and friends, it continues to make a difference!

Thanks to ALL of you, this money goes directly to fund research powered to rapidly develop treatment for KAND. We could not advance our mission without the community of families and supporters surrounding us. Thank you! Did you miss our Rare Disease Day fundraiser and still want to donate towards our mission? No problem! Click here to donate. Thank you for supporting our family-led organization.

KIF1A.ORG’s Therapeutic Development Strategy Series: Video 1

“Overview of Therapeutic Development Strategy” is the first in a series of videos presented by our Chief Science Officer, Dr. Dominique Lessard, to comprehensively review KIF1A.ORG’s therapeutic development efforts. Video 1 walks through how we drive therapeutic development forward, our strengths as an organization, the importance of risk and failure in therapeutic development, and more! Be on the lookout for our next video which will describe our therapeutic development pipeline, different types of therapeutic targets, and current projects that are in various stages of development.

KIF1A.ORG’s Small Molecule Drugs 101 Video

What are small molecule drugs? Where do they come from? How do they work? This video and accompanying blog post was created by a team of biotechnology students at Columbia University as part of a class project for the Seminar in Biotechnology taught by Dr. Lili Yamasaki. Thank you to Yanzhe Ma, Yishu Yin, Jerry Shen, Alex Jin, and Wanyun Li for volunteering your time and expertise to create this Research Simplified resource for the KIF1A.ORG community!

KIF1A In The News

Hoyt’s Story Featured by Good Morning America

Hoyt’s family shares how they’ve been impacted by his recent KAND diagnosis and their hopes for future research leading to treatment with Good Morning America!

Emma’s Family Shares Their Story

Since receiving Emma’s KAND diagnosis this month, her family has eagerly joined our community’s efforts to spread awareness and raise funds. They have since shared their story through Canadian news outlets, and CBC News Radio!

KIF1A Superhero Emma with Family

Whatever role you play in our community, we need you. Thank you for all that you do to help us realize our mission for KIF1A superheroes everywhere.


  1. 1
    Anne-Marie Finnell on March 30, 2021

    Thank you for keeping us informed!

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