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Randal Kaufman included in $12 million initiative to improve hemophilia treatment

The new project will help researchers better understanding why current gene therapy treatments aren't working.

Blog

Rare Disease Day gathers scientists, doctors and families

After a break during the pandemic, Rare Disease Day is back and better than ever.

Blog

Rare disease in the time of COVID: Damian Omler’s story

How a one-of-a-kind kid and his family stay connected during the pandemic.

Paper of the Week

A FBXO7/EYA2-SCFFBXW7 axis promotes AXL-mediated maintenance of mesenchymal and immune evasion phenotypes of cancer cells

Blog

One at a time: How a Sanford Burnham Prebys professor changes patient lives

Having worked for decades to improve the lives of children with rare diseases, Hudson Freeze is still on the case.