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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.

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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. 

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Resources for Parents and Caregivers

Resources for patients who have a precision diagnosis.

Resources for patients who are still undiagnosed.

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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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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.

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