In its latest effort to find a cure for Duchenne muscular dystrophy, CureDuchenne has released a centralized data platform that connects patient data and biosamples with scientists and drug developers.
Duchenne is the most common form of muscular dystrophies, a group of muscle-wasting genetic disorders that can lead to the failure of heart and breathing muscles. It impacts an estimated 300,000 boys around the world.
The Newport Beach-based nonprofit began enrollment for CureDuchenne Link on July 9.
“With the launch of CureDuchenne Link, we hope to break down existing information silos and offer qualified researchers access to participant data and biosamples in one all-encompassing platform,” Chief Executive Debra Miller said.
A key function of the platform, in development for two years, is a profile that shows genetic information, medical records, biosamples and more for each participant in one convenient place; the participants are anonymous.
The platform was self-funded with a $1 million investment, and will be freely available, unlike other data platforms and biobanks that have exclusive relationships with universities or pharmaceutical companies, she pointed out.
Both Duchenne and Becker muscular dystrophy “are complex diseases with thousands of mutations. To effect real change, we need research into every one of those mutations,” Miller said.
Debra and her husband, Paul, formed CureDuchenne in 2003 shortly after their son, Hawken, was diagnosed with Duchenne.
Debra, a Business Journal Women in Business Award honoree in 2015, adopted a venture philanthropy business model to pursue a cure, and has successfully raised over $100 million from pharmaceutical companies to fund Duchenne research.
