Isabella Coulstock had never performed Who I Am live before that night in Paris.

When she finally did, the room went still.

As her voice filled the space, a video played — a montage of Josie's life, her happiness, her hard times, the big heart that takes her mommy's hand. FOXG1 families felt seen. Those newer to the cause felt the door open wide.

Many were in tears. All of them reminded, in the span of a few minutes, exactly why this work matters — and who it is for.

Rare Disease Day at the University at Buffalo reminded us of something we never want to take for granted: what becomes possible when science, urgency, and mission are truly aligned.

From Dr. Soo Lee's presentation on the foundational science behind our AAV9 gene therapy, to Gai Ayalon's candid look at the complex journey from academic discovery to clinical-stage therapeutic, to the inspiring poster session showcasing the depth of research underway in the Lee lab — the day was a powerful testament to what a community united around a common goal can achieve.

FOXG1 families and Foundation leadership visited the Rockville CDMO site at Charles River Laboratories — where our gene therapy is being manufactured.

We saw firsthand the precision, safeguards, and care behind plasmid and AAV vector production, and the responsibility carried at every step.

We are deeply grateful for this partnership and the commitment to bringing treatment to children with FOXG1 syndrome.

The FOXG1 Research Foundation has named Brandon M. Henry, M.D., as Chief Medical Officer as FRF-001 advances toward a first-in-human clinical trial. Dr. Henry brings deep experience in AAV gene therapy development and will lead clinical strategy and oversight as we prepare to move into patient clinical trials.

As a parent-led organization serving as the independent sponsor of a multi-site, international gene therapy trial, FRF is pioneering a new rare disease model—advancing treatment with urgency while upholding the highest scientific and regulatory standards. Dr. Henry’s leadership strengthens our path forward as we work to bring a potential treatment to children and families impacted by FOXG1 syndrome.

The FOXG1 Research Foundation announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to FRF-001 for the treatment of FOXG1 syndrome — marking another major regulatory milestone as we advance toward our first-in-human Phase 1/2 clinical trial.

The FOXG1 Research Foundation announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application to begin first-in-human clinical trials for FRF-001, the first FOXG1 AAV9 gene replacement therapy. This historic milestone marks the first instance of a parent-led rare disease nonprofit foundation independently sponsoring its own multi-site, international gene therapy clinical trial.

2025 marked a pivotal year for the FOXG1 Research Foundation.

We successfully completed IND-enabling toxicology studies and submitted an Investigational New Drug (IND) application to the FDA, advancing the first-ever FOXG1 gene therapy toward patient clinical trials in 2026.

As a global, parent-led organization, we continue to demonstrate that families can independently drive rare disease drug development—executing clinical trials with rigor, urgency, and responsibility.

Alongside this progress, we expanded patient care, research, advocacy, and global awareness, while honoring the lives lost within our community and reaffirming why this work cannot wait.

Together with our families, donors, scientific partners, and clinicians, we are building a future where treatment is possible.

During FOXG1 Awareness Month, our FOXG1 got the Luckiest gift - to see one of our own featured on ESPN!

Lawson Luckie - one of the most sought after tight end prospects in the country coming out of high school in Norcross, Georgia - had an opportunity to play almost anywhere, but only one school provided him the opportunity to be something that the others couldn’t.

As much as Luckie could’ve been the Big Man on Campus, he’s always prioritized an even bigger role, as a big brother to Cannon. It’s a relationship built on love and loyalty that has the Georgia tight end living his life with far more than wins and losses on his mind.

Published in the Journal of Neurodevelopmental Disorders, this peer-reviewed study presents the largest and most detailed analysis to date of FOXG1 syndrome. The results come from the FOXG1 Citizen Health Natural History Study, led by the FOXG1 Research Foundation’s Chief Clinical Data Officer Elli Brimble, in collaboration with Citizen Health and international key clinical partners.

The FOXG1 Research Foundation is proud to share that FOXG1 syndrome has officially been granted its own ICD-10-CM code — QA00151 — by the CDC, effective October 1, 2025. This milestone, years in the making, gives FOXG1 families long-overdue visibility in the healthcare system. With a dedicated code, clinicians can record FOXG1 in medical records, insurers have stronger grounds for coverage, and researchers can more accurately track patients — a powerful step forward for our community and for rare disease recognition.

The FOXG1 Research Foundation announced the FDA has granted Orphan Drug and Rare Pediatric Disease designations to FRF-001, its lead gene therapy for FOXG1 syndrome. FRF-001 marks a historic first: a parent-led foundation sponsoring its own international, multi-site clinical trial. These designations provide regulatory and financial incentives that help advance FRF-001 toward patients, underscoring FDA’s recognition of this parent-driven effort.

My firstborn son, Crosby, was diagnosed with FOXG1 syndrome. He was just six months old when we got the news. Since then, every night, every weekend, and every ounce of spare energy I’ve had has gone into fighting back against the silence and suffering that FOXG1 imposes on children like my son.

Today, I have something different to share: hope. We’re preparing to bring a first-of-its-kind AAV9 gene replacement therapy for FOXG1 syndrome into the clinic in 2026. That’s not a hypothetical. It’s real. It’s happening. And it could change everything.