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 neurodevelopmental and neurodegenerative conditions that 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.

Project Baby Lion

Resources for Parents and Caregivers

Resources for patients who have a precision diagnosis.

Resources

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.

News & Research

In little Leo, the face of future personalized medicine

A California biotech has leveraged artificial intelligence and other cutting-edge tools in a perilous rescue mission focused on a 2-year-old Setauket boy born with an unusual genetic disease.

STAT News: Can AI build custom ‘n-of-1’ drugs faster?

Two years ago, Yiwei She was searching desperately for someone to build a drug for her newborn son, Leo. Leo was born with a very small head, a symptom of many severe diseases. After a seizure at two months, doctors sequenced his genome and found a single misspelling in a gene called TNPO2.

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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.

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