Advancing FOXG1 Research with Hope for a Cure.
FOXG1 Research Center is dedicated to understanding and finding treatments for FOXG1 syndrome and other neurodevelopmental disorders.
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What is FOXG1 Syndrome?
FOXG1 syndrome is a rare neurodevelopmental disorder caused by mutations in the FOXG1 gene, crucial for early brain development. It affects approximately 2.8-3.5 per 100,000 live births worldwide, with over 1,000 identified cases to date.
Children with FOXG1 syndrome may experience a range of symptoms, including brain abnormalities, epilepsy, movement disorders, and features of autism spectrum disorder. The FOXG1 gene is key in developing the cerebral cortex, responsible for cognitive functions, motor skills, and sensory processing.
FOXG1 syndrome profoundly impacts physical and cognitive abilities. However, ongoing research is focused on understanding its mechanisms and developing treatments to improve the quality of life for those affected.
Meet Yuna, now 14 years old, but captured here at 4 in a moment that reflects her joyful spirit. While FOXG1 syndrome presents daily challenges, such as difficulty with communication and movement, Yuna’s radiant smile and love for her family speak louder than words.
Leading the Charge in FOXG1 Research
Drs. Soo and Jae Lee, a husband and wife duo, lead the FOXG1 Research Center. Their mission to cure FOXG1 syndrome is personal—their daughter, Yuna, was diagnosed with this rare genetic disorder, inspiring their dedicated research and pursuit of effective treatments.
Together with a team of passionate and dedicated professionals, they are committed to making breakthroughs that will bring hope to families affected by FOXG1 syndrome.
The Path to a Cure in Conjunction with FOXG1 Research Foundation :
Our Roadmap
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Patient specific pluripotent stem cell models (FOXG1 patient iPSCs)
Mouse Models (covering each FOXG1 mutation type)
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Understand the impact of FOXG1 mutations on the brain and body: molecular, cellular and behavioral analysis
Identify robust biomarkers and endpoints
Learn what time-points are effective for treatment
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Identify and screen various therapies including: Gene Therapies, ASO's, RNA, etc.
Screen approved drugs
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Conduct preclinical (animal) efficacy and toxicology studies on candidate molecules that passed the screening stage.
Submit Investigational New Drug application to FDA and other regulatory agencies.
Obtain approval to progress to clinical trials in human patients.
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Conduct clinical trials to assess the safety and efficacy of the investigational new drugs
Seek regulatory agency approval based on the outcome of the clinical trials
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Bring the community new treatments for FOXG1 syndrome
Make an impact
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Make an impact ·
Your Support Fuels Our Mission
Donations play a critical role in advancing the research at the FOXG1 Research Center. Every contribution directly funds the groundbreaking studies being conducted in our lab, helping us move closer to finding effective treatment options and ultimately a cure for FOXG1 syndrome.
Your generosity empowers our dedicated team of researchers to continue their vital work, bringing hope to families and transforming the future for those affected by this rare genetic disorder.
We work closely with FOXG1 Research Foundation. All donations can be made directly to FRF to support our efforts.
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