News and Trends 20 Jul 2022
Upstaza gets nod from Europe for AADC deficiency treatment
PTC Therapeutics, Inc. says Upstaza (eladocagene exuparvovec) has been given marketing authorization by the European Commission. Upstaza is the first approved disease-modifying treatment for aromatic L-amino acid decarboxylase (AADC) deficiency, and the first marketed gene therapy directly infused into the brain. It is approved for patients 18 months and older. “Today’s approval from the European […]