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.
News & Research
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.