Imagine if we could change the fate of KIF1A-Associated Neurological Disorder, a progressive—sometimes fatal—neurodegenerative disorder. Instead of a doctor telling parents there’s nothing they can do to help or save their child, we envision a future where doctors can say “It’s going to be okay. There is treatment.”

This Is How We Fast-Track Studies on Promising Treatments

KIF1A.ORG is a community of families, scientists and supporters like you who are funding projects to find treatment for our superheroes. From small molecule drug discovery to antisense oligonucleotide therapy, we’re working rapidly to outpace the ticking KIF1A clock. Our superheroes have no time to wait. Every time a child has a debilitating seizure, requires invasive surgery, or loses their life, we are reminded that we MUST move faster.

The good news is that KIF1A.ORG and the KIF1A Research Network have already identified therapeutic candidates that show promise to treat KAND. These therapeutics have already been developed for related diseases such as ALS, hereditary spastic paraplegia and Charcot-Marie-Tooth disease. The problem is we won’t know whether these candidates can treat KAND until we do the preclinical work to prove their effectiveness.

That’s why we’re launching the Treatment Accelerator Program (TAP) to expedite the search for treatments for our superheroes. TAP will enable KIF1A.ORG to hire a Contract Research Organization (CRO) to design and manage a system to model KAND in cells and test potential therapeutics for KAND. CROs are specialized labs that pharmaceutical companies use to execute research and development. By leveraging their services, KIF1A.ORG can move at scale and speed to expedite our path to the world’s first clinical trials for KAND.

You Can Make an Impact

Help KIF1A families drive therapeutic development by making a donation today. This program will add to our Tools for Development that are freely available to the scientific community, including pharmaceutical and biotechnology companies. These tools require significant time and resources, and are often the reason research institutions and companies choose not to invest in developing treatment for rare diseases. Not with KIF1A. With our accessible tools, we lay the groundwork for fast and efficient therapeutic development.

$100,000+ of $100,000 USD Raised as of May 13 to Launch Treatment Accelerator Program

May 13 Update

Friends, we did it. In less than a month, we raised $100,000+ USD to launch the Treatment Accelerator Program to expedite the search for treatments for our superheroes. With your help, we WILL change the fate of KAND. Thank you for every donation, every share and every note of encouragement. If you’d like to donate to help us do even more, every donation goes directly to KIF1A research.