From Determination to Progress: Celebrating 7 Years of the FOXG1 Research Foundation

Seven years ago, a group of parents huddled together in a small conference room, united by a common goal: to drive a cure for our children diagnosed with FOXG1 syndrome. We didn't have any scientific expertise. We didn't have assets like mouse models or cell lines, and we certainly didn't have money. What we did have was determination, love for our children, and an unwavering belief in the power of parents.

From those humble beginnings, we have shown that the power of parents is tremendous. Over the past seven years, we’ve built an incredible range of assets. We’ve organized a community that was once scattered and isolated, creating a collective force for progress. We launched a digital natural history study on Citizen Health that includes over 770 cumulative patient data years, enabling us to select clinical endpoints and understand our patient population without burdening our patients with site visits. We've established a FOXG1 Center of Excellence, laying the foundation for a generation of new drugs. Most excitingly, we have identified a promising gene therapy that we are working to bring into clinical trials.

But we aren’t stopping there. We are also pioneering a blueprint to reduce the cost of creating a drug for an ultra-rare disease from an estimated $180 million to around $22 million. This is about more than just FOXG1 syndrome; it's about setting a new standard for how we approach rare diseases.

The journey has been hard, filled with emotional highs and lows, but it has undoubtedly been the most worthwhile endeavor of our lives. None of this would be possible without the hundreds of caregivers who have banded together, driven by love and a fierce determination to fight for our children, and the countless advisors who have selflessly given their time and expertise to guide us.

Together, we are proving that parents can drive monumental change. We are not just waiting for a miracle; we are creating one.