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.

New FOXG1 Research publication in Nature Communications. Title: The patient-specific mouse model withFoxg1frameshift mutation provides insights intothe pathophysiology of FOXG1 syndrome

The FOX DEN - March Issue is here with exciting updates for the FOXG1 community! Join our FOXG1 Advocacy Team, celebrate news from the FOXG1 Research Center, and support fundraising efforts for gene therapy. Plus, don’t miss inspiring stories from FOXG1 families making an impact worldwide! Read more.

2024 marked seven years of the FOXG1 Research Foundation. We are a parent-driven, global foundation with the mission to improve the lives of every person affected by FOXG1 syndrome worldwide. We are immensely proud of the organization we have built and the resources we have made available to our community since 2017, but we could never have come this far without our incredible donors and community. Please read the impact of the support in 2024 here.

We kicked off 2024 with a milestone achievement: our CEO was honored as the opening speaker at the inaugural White House Rare Disease Forum. This invitation reflects our growing reputation as leaders in the rare disease space and served as a powerful platform to advocate not only for FOXG1 syndrome but for the unmet needs of all rare diseases worldwide.

Read our 2024 Impact Report here…

Modifying DNA was supposed to be a game changer. But drug companies and Wall Street are pulling back from the expensive-to-produce treatments, leaving parents to fund research on their own. When a biotech company promised to study a new treatment for her daughter’s disease, Nicole Johnson was elated. She’d been raising money to research potential cures for FOXG1 syndrome, a rare neurological disorder that had robbed her daughter of the ability to walk or talk. A Dallas-based biotech, Taysha Gene Therapies Inc., agreed to collaborate with her nonprofit on developing a gene therapy for the condition.

But two years ago, Taysha stopped developing the treatment and more than a dozen others. The decision was hardly unusual. Since 2021, drug companies have dropped more than 50 gene therapies. The rising number of abandoned treatments raises questions about whether modifying a patient’s DNA to cure rare diseases can be a lucrative business. And it’s sparked an effort from parents, scientists and entrepreneurs to rescue the drugs that have been left sitting on the shelf.

Hope and love aren’t often mentioned in the same breath as scientific research, but they’re exactly what UB’s FOXG1 Research Center (FRC) was founded on. 

“Love for every individual living with FOXG1 syndrome and other neurodevelopmental disorders, as well as their caregivers, and hope that we can find a cure for FOXG1 syndrome and related disorders on the basis of scientific discoveries we are making,” Soo-Kyung Lee, director of the FRC and the parent of a child with FOXG1 syndrome, told a crowd gathered to celebrate the center’s official opening Tuesday. 

WILMINGTON, Mass.--(BUSINESS WIRE)--Jul. 30, 2024-- Charles River Laboratories International, Inc. (NYSE: CRL) announced today a collaboration with the FOXG1 Research Foundation (FRF) highlighting the patient advocacy group’s model to independently drive drug development through the clinical phase. The parent-led global organization driving the research to cure FOXG1 syndrome and related neurological disorders will collaborate with Charles River in a comprehensive gene therapy contract development and manufacturing organization (CDMO) agreement.

“Charles River is proud to work with the FOXG1 Research Foundation to advance its gene-therapy through clinical trials,” said Kerstin Dolph, Corporate Senior Vice President, Global Manufacturing, Charles River. “The FOXG1 patient population has an incredible unmet need, and we are looking forward to lending our expertise to FRF as they continue to trailblaze a path toward providing rare disease treatments.

Groundbreaking Study Shows Promise in AAV9 Gene Therapy for FOXG1 Syndrome; Rescue of Brain Structure Abnormalities and Deficits.

[Buffalo, New York June 10, 2024] – A landmark study led by Dr. Soo-Kyung Lee, Chief Scientific Officer at the FOXG1 Research Foundation and Empire Innovation Professor and Om P. Bahl Endowed Professor in the Department of Biological Sciences at University at Buffalo, and Dr. Jae Lee, Professor in the Department of Biological Sciences at University at Buffalo, in collaboration with Dr. Kathrin Meyer (responsible for the SMA gene therapy), has been published in Molecular Therapy Methods & Clinical Development.