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.

Rare Disease Day gathers scientists, doctors and families

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

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.

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.