“In half of the cases, the diagnosis has led to changes in treatment management ... In addition, patients with rare diseases are “naturally occurring single-gene knockout humans ...

It’s a Schaumburg mother’s mission to find a cure for a rare disorder that affects ... “We have to try every single possible way, because the kids’ lives are dependent on this.” ...

WOODBRIDGE, Conn.--(BUSINESS WIRE)--Cure Rare Disease (CRD) announced it has been awarded a $5.69 million grant from the California Institute for Regenerative Medicine (CIRM) to advance the ...