The FOXG1 Gene Therapy is Within Reach!

The FDA has cleared the FOXG1 gene therapy to begin patient clinical trials.

We’re making history.

This is parent-led drug development in action— bringing the first treatment for FOXG1 syndrome to children around the world.

Help Us Get There

Learn about FOXG1 Gene Therapy

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Smiling young boy with curly hair in a green t-shirt sitting on a wheelchair, outdoors in front of a brick wall.

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A smiling girl in glasses sitting in a motorized wheelchair indoors, with toys and a person standing nearby.

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A young child with blue eyes, wearing red glasses, a gray cap decorated with sports and animal patterns, and a white bandana with red and black designs, sitting in a backpack carrier outdoors among trees.

A baby with blue eyes and light brown hair, wearing a white outfit with a bunny embroidery, lying in a decorated crib with pink and white patterned bedding.

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A young girl with a big smile, showing her teeth, with dark hair tied back and wearing a floral shirt.

A smiling young boy with glasses and missing front teeth.

A young boy with blonde hair and blue goggles sitting outdoors on a blanket, wearing a gray hoodie and blue pants, holding a teething toy in one hand and touching his mouth with the other.

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What is FOXG1 Syndrome

Illustration of a DNA double helix with a break and red arrows indicating a mutation or damage.

A young red-haired boy who has FOXG1 syndrome with blue eyes sitting on green grass, looking up and smiling.

FOXG1 syndrome is a rare neurological genetic disorder that impacts early brain development and typically causes an array of disabilities and medical complexities, including epilepsy. Many children with FOXG1 syndrome cannot walk or talk or take care of their basic needs. Mutations to the FOXG1 gene are mostly non-inherited (de-novo).

There are currently about 2000 known people in the world diagnosed with FOXG1 syndrome. We're living at a pivotal moment for FOXG1 syndrome. The first treatment is advancing through patient clinical trials.

About FOXG1 Syndrome

The FOXG1 Research Foundation

Dedicated to Cure FOXG1 Syndrome

The FOXG1 Research Foundation (FRF) is the parent-led, global organization driving the research to cure FOXG1 syndrome and related neurological disorders, while supporting and advocating for patients and families worldwide.

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About the FOXG1 Gene Therapy Campaign

Making Treatments Possible, Together

It takes a dedicated community to advance FOXG1 treatments. Whether you’re a parent seeking answers, a donor ready to drive change, or a scientist exploring new frontiers, your role is critical in shaping the future.

Parents & Caregivers

Learn about resources, studies, & support

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Donors & Philanthropists

See ways to support, impact, campaign, and more

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Researchers & Industry

Science, partnerships, clinical trial

Read Bio

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Newly Diagnosed?

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You are not alone, and there is real hope on the horizon.

A treatment for FOXG1 syndrome is no longer a dream — it is becoming a reality. We are here to walk this journey with you.

Start your Journey with Us

Register Your FOXG1 Child Here

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FOXG1 Parents: The most important thing to do right away, is to register your FOXG1 Child in the official FOXG1 syndrome patient registry.

This helps advance treatments for all patients worldwide.

The de-identified information you share in the global registry as well as through a Citizen Health account is the most powerful way to deepen the understanding of FOXG1 syndrome and help the entire FOXG1 community.

Register Your FOXG1 Child

Create Your Citizen Account