From a parent’s promise, to a global movement changing the landscape of rare disease treatments.

Our Story

A Promise to Every Child

When our children were diagnosed with FOXG1 syndrome, we were told there was nothing we could do. No treatments, no concerted research underway, no roadmap. Just a devastating diagnosis for a disorder few had ever heard of.

But we refused to accept that. 

In 2017, parents from around the world connected over a Skype call. That conversation became a movement—and the start of something historic.

We began with a promise: to work toward a healthier life for every child in the world born with FOXG1 syndrome—no matter their gene variant, gender, or location. That promise was rooted in our determination to be innovative, to challenge what was possible, and to forge new paths where none existed. It remains the driving force behind everything we do.

From that moment to today, we have gone from having no path forward to advancing the first-ever treatment for FOXG1 syndrome: a promising gene therapy now being brought to children around the world. We founded the FOXG1 Research Foundation—a bold, parent-led organization driven by the belief that love, science, and urgency could change the future.

Nicole Zeitzer Johnson sitting on a couch, smiling, with her daughter Josie, in a blue shirt sitting on her lap, in a cozy living room.
Nasha Fitter, co-founder of FOXG1 Research Foundation wearing a hat and a colorful top holding her daughter Amara who is wearing a floral dress outdoors.
A woman lying in bed with a young child, sleeping next to her, both covered by a blanket.

From Isolation to Innovation

We knew from the start that to help every child, we needed a scientific strategy that reflected the full range of FOXG1 variants. We funded and developed the world’s first suite of FOXG1 mouse models and stem cell lines, representing the varying mutations. We built powerful AI-powered patient data platforms to deeply understand the relationship between each child’s genetic variant and their symptoms. We made research tools and biobanks accessible to support FOXG1 science around the world.

From a place of complete isolation, we launched a global community. Families from more than 50 countries now stand together, united by the mission to accelerate treatments, bring forward a cure, and support each other every step of the way.

Three women smiling at a conference, wearing black blazers and orange lanyards.
A group of five women sitting around a table in a cafe, engaged in a discussion or working together. The cafe has purple walls decorated with various framed paintings and artwork.

A Strategic Path to a Cure

Guided by a world-class Scientific Advisory Board of neurologists, geneticists, clinicians, and BioPharma leaders, we built a strategic roadmap to a cure. We assembled a translational science team to advance gene therapies, antisense oligonucleotides, small molecules, and emerging therapeutic technologies.

To accelerate clinical readiness, we launched the FOXG1 Patient Data Center—a global digital ecosystem made up of the FOXG1 Patient Registry, a pioneering Digital Natural History Study (supported by the Chan Zuckerberg Initiative), and the FOXG1 Stem Cell Biobank. We made these tools accessible to researchers everywhere.

We held our first FOXG1 Scientific Symposium in 2018 and have since hosted multiple symposiums and two global parent conferences—bringing together scientists, clinicians, and families from over a dozen countries.

In 2024, we celebrated a major milestone: the official ribbon cutting signifying the opening of the FOXG1 Research Center at the University at Buffalo. The Lee Lab at the center—led by two of the world’s leading neuroscientists studying FOXG1 syndrome, who are also the devoted parents of Yuna, a young teenager with FOXG1 syndrome—published groundbreaking research that supports the path for our gene therapy program. In one of the most extraordinary coincidences imaginable, the Lees were studying the FOXG1 gene long before Yuna was born. This study confirmed what we believed: the science is ready.

Group of diverse people posing for a photo at a conference with a backdrop displaying the FOXG1 Research logo and website.
A group of people standing together in front of a blue informational backdrop for the FOXG1 Research Center at University at Buffalo. They are holding a blue ribbon that reads 'University at Buffalo FOXG1 Research Center' and smiling for the photo. There are balloons on the left side.

The FOXG1 Gene Therapy Program

Today, we are making history. We are the first rare disease foundation to independently drive a gene therapy program through global, multi-site clinical trials. Learn more about our groundbreaking FOXG1 gene therapy program here.

Leading a Movement for Rare Disease Innovation

As parents, we put our heads down and did everything we could to create a healthier life for children with FOXG1 syndrome. But along the way, our work began to create ripples far beyond our community. We realized we weren’t just pushing forward for FOXG1—we were helping reshape the landscape of rare disease drug development as a whole.

With support from the Chan Zuckerberg Initiative, we pioneered a novel AI-powered digital model for natural history studies—an approach that is now being used by more than 100 other rare disease groups to better understand their patient communities. We’ve become recognized leaders in rare disease innovation—mentoring other patient organizations, pioneering real-world data platforms, and building a blueprint that could accelerate treatments across diseases.

Group of five people standing behind large white letters spelling ASGCT at an Annual Meeting event in 2024 in Maryland, with a backdrop displaying event details and a sunset sky.
A woman and a young girl playing with blocks and toys indoors near large windows with greenery outside.
A woman scientist and two men scientists in lab coats explaining DNA structure on a whiteboard
A young boy with cancer sitting in a stroller, laughing and holding hands with a man and a woman outdoors, with the woman gently touching his head.

A New Blueprint for Rare Disease Drug Development

We are proving the power of parent-led drug development—driven not by profits, but by the return on investment that matters most: the lives and futures of children.

As Yale Weiss of Mahzi Therapeutics says, the FOXG1 Research Foundation is "radically transforming the rare disease drug development landscape."

We have built a replicable model for rare disease treatment development that drastically reduces the time and cost of drug development —without sacrificing scientific rigor.

Rare Disease Advocates

In 2023, our co-founder Nasha Fitter took the podium as the first speaker at the inaugural White House Rare Disease Forum. You can watch her speech here. She spoke on behalf of all 300 million people living with a rare disease worldwide—half of whom are children—advocating for urgent change and a new way forward.

A Global Community of Love, Science, and Action

We are more than a research organization—we are a community. We are a patient advocacy group. We provide critical resources, webinars, and support groups for caregivers and families. We stand beside families in their darkest hours, even helping rescue and evacuate a FOXG1 family from Ukraine during wartime.

We now have chapters in France, Italy, Spain, Australia, Japan, and more launching. We are building bridges across borders, across disciplines, and across sectors—uniting parents, clinicians, scientists, philanthropists, and biotech to accelerate hope.

As co-founder Nicole Zeitzer Johnson says:

“We are motivated by every child, the incredible breakthroughs in science, the strength of our community, and the firm belief that nothing is impossible. We know that this work we set out to do—motivated by our love for our children—will benefit children all around the world with FOXG1 syndrome and will change the course of this disease for children who are not even born yet.”

We are building the future. And we invite you to be part of it.

A woman with glasses is sitting at a desk in a workspace, smiling at the camera. She is operating a computer with a large monitor displaying brain scan images. The workspace has multiple computer screens and equipment, with a blue cover or bag in the background.
A happy family of four standing outdoors in a garden during sunset. The mother and father are smiling and looking at each other, holding their two young children, a girl and a boy, who are also looking at the camera.
Three women smiling at a FOXG1 Research conference, wearing black blazers and orange lanyards with name tags, indoors with a window in the background.
Image of Nasha Fitter from FOXG1 Research Foundation speaking at a podium at the White House with an American flag in the background. The text on the screen identifies her as Nasha Fitter, FOX61 Research Foundation's cofounder and CEO.
A group of people at the University at Buffalo FOXG1 Research Center ribbon-cutting event at the University of Buffalo, standing in front of a digital screen displaying the center's logo, with balloons on the left side.
Group of nine people standing in front of a FOXG1 Foundation banner at an event, smiling and posing for the photo.
Five women sitting around a table in a cafe or restaurant, engaged in conversation and reading. The wall behind them is decorated with various paintings, including a large black and white horse's head and smaller abstract and floral art.
A woman standing next to a large pink promotional poster for the book 'Joyfully Josie' by Nicole Zeitzer Johnson. The poster features animated children playing and the book's title and website information.
Group of seven people, including a young boy in a wheelchair, smiling inside a hotel lobby.
Two smiling women standing in front of a welcome sign at a FOXG1 Science conference, with lanyards and badges.
A conference room with a stage and a presentation screen showing two questions related to clinical trials. There are three speakers on the stage, one woman standing at a podium labeled FoxG1 Research Foundation, a woman seated on the stage, and a man
Group photo of diverse professionals at a conference, standing in front of a backdrop with the FOXG1 research logo.
Group of five people attending the ASGCT 2024 Annual Meeting in Maryland, standing in front of a large event sign with large white letters spelling 'ASGCT' and a backdrop with the event logo.
Two women standing outside of the NIH building with a glass entrance, smiling at the camera. One woman is wearing a black jacket and carrying a large red handbag. The other woman is wearing a beige coat and holding a coffee cup.
Image of Nasha Fitter, guest speaker, smiling, with a background of medication pills and a pill bottle, promoting a discussion on the importance of rare disease research.
Family and friends sitting on grass at an outdoor gathering, smiling for the camera, with a white tent in the background.

FOXG1 Research Foundation Timeline