Notizie in inglese per livelli

A New Gene Therapy Reaches the Root Cause of a Rare Liver Disease for the First Time

The US Food and Drug Administration granted accelerated approval to GENGLYCOS, a one time gene therapy for glycogen storage disease type Ia, a rare inherited disorder that leaves patients unable to safely release stored sugar from the liver. The therapy, developed by Ultragenyx and based on a Phase 3 trial that showed a significant drop in patients' need for constant cornstarch dosing, is designed to treat the disease's underlying genetic cause rather than just managing its symptoms.