A Courageous Future for Ultra-rare Children
We’re reshaping ultra-rare care to support all children and the families who love them — starting with those who need it most.
Forging New Pathways for Precision Medicine
In the U.S. alone, millions of children live with an ultra-rare disease.
240,000 of those children suffer from a neurodegenerative disease.
Up to a million children suffer from a severe neurodevelopmental condition.
These are treatable with precision medicine.
Yet our complex medical system abandons ultra-rare children and their families to navigate this biomedical wilderness alone, offering little guidance after a devastating diagnosis — or no diagnosis at all.
The time to act is now. The science, technology, and willpower to bridge these systemic gaps is at our fingertips. With measured optimism and a growing body of revelatory science, we’re building a sustainable future of personalized medicine that is accessible to everyone.
Project Baby Lion
Rallying To Rescue Ultra-rare Infants and Children
Diagnosis is just the first step for an ultra-rare child and family. Project Baby Lion leverages emerging technology to develop individualized medicine and bridge the gaps for the therapeutic odyssey.
Resources for Parents and Caregivers
Resources for patients who have a precision diagnosis.
Resources for patients who are still undiagnosed.
Our Partners & Collaborators
We partner with visionary organizations from every area of the biomedical landscape, from academic researchers to institutional funders who want to make a world of difference.
- More to Come
News & Research
On a Medical Frontier, Parents Push Forward
Four years on from Leo’s diagnosis, a profile of what it cost to build him a medicine — and of the NICU sequencing pilot the foundation has since run at Stony Brook.
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Yiwei She honored as 2025 Woman of Distinction
New York State Senator Anthony Palumbo names Yiwei She the First Senate District’s Woman of Distinction, for the research she drove after Leo’s diagnosis and for founding the TNPO2 Foundation.
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Donate
Our work relies on collaboration and generosity. Join the researchers, clinicians, funders, policy-makers, and caregivers who are creating new models of care for ultra-rare patients.
