BUFFALO, NY – Five major research centers in New York have banded together to share resources and a manufacturing facility in ...

The firm will explore FORX-428's activity in patients whose tumors harbor DNA damage repair mutations or mutations that cause high DNA replication stress.

Kumquat, based in San Diego, received US Food and Drug Administration clearance of its investigational new drug application for the KRAS G12D inhibitor in July. The firm will be responsible for ...

In the EMPEROR trial, children with a confirmed variant in the SCN1A gene not associated with gain-of-function mutations will receive zorevunersen or a placebo.

The Rare Therapies Launch Pad pilot aims to create a streamlined path to develop individualized treatments and get them approved.