People with erythropoietic protoporphyria (EPP) must go to great lengths to avoid sun exposure. There have been no approved treatments that address the disease mechanism, but a new drug could change that.
Forty years after the Orphan Drug Act passed, researchers advance drug development for neglected rare conditions everywhere from the lab bench to backstage.
Explore how emerging in vitro systems — built from primary cells, cocultures, and vascularized tissues — are improving translational research outcomes.