Phase One: Finding the Fastest Disease-Modifying Options

Raised: $350k - Target: $500k

Living with CDKL5 Deficiency Disorder (CDD) means living in a constant state of emergency. Every parent of a child with refractory epilepsy knows how devastating it is—many of us spend weeks or months in the hospital. We do not have the luxury of waiting for theoretical academic research or decade-long timelines for novel drug discovery and gene-correcting treatments to become available.

We need meaningful treatments today.

The fastest path to treatment is to explore existing, market-available drugs that may help—a process known as drug repurposing. To do this effectively, we must turn every stone and exhaust every possible avenue in search of treatments that can make a difference now.

Phase Two: Advancing Discoveries and Investing in Translational Research

Raised: $0 - Target: $5M

Phase One will yield insights into promising drug candidates and uncover new potential therapeutic targets. Some of these candidates will require further research, leading to molecular refinement and potential new Investigational New Drug (IND) applications. Additionally, understanding their mechanisms of action may unlock entirely new treatment possibilities. In Phase Two, we will invest in both expanding promising leads from Phase One and pursuing novel approaches, including:

1. AI-driven drug discovery
2. Next-generation gene-correcting technologies
3. Non-viral delivery mechanisms for redosing therapies

By supporting this phase, you are fueling breakthroughs that extend well beyond CDD and into the broader field of neurological disease treatment.

Why donate?

CDD is uniquely suited for therapeutic advancements because it is:

1. Monogenic (caused by a single gene mutation, making it a clear target for intervention)
2. Well-researched (offering a strong foundation for rapid progress)
3. Reversible (meaning that, with the right treatment, symptoms could be dramatically improved or even eliminated)

Without disease-modifying therapies, affected children endure lifelong severe disability and suffering. But beyond compassion, supporting CDD research can have a transformative impact on medicine as a whole. Validating treatments in rare monogenic disorders like CDD sets the stage for broader applications to Alzheimer’s, Parkinson’s, and other neurological diseases. Even if CDD does not directly affect you, investing in its cure may one day save you or someone you love. Your support today is an investment in a healthier, more equitable future for all.

How you can help

Due to laws regulating donations internationally and domestically within the US, we are currently only able to accept donations from the following states:

1. California
2. Connecticut
3. New Jersey
4. New York
5. Texas

We are working on expanding this list, but meanwhile, if you want to contribute to our mission in other ways, please contact us.

We are a 501(c)(3) non-profit organization, and all donations are tax deductible.