Ultragenyx Wins Accelerated Approval for Genglycos, First Gene Therapy for GSDIa
The FDA granted accelerated approval to Ultragenyx’s Genglycos (pariglasgene brecaparvovec-opnr; DTX401), the first approved treatment for glycogen storage disease type Ia (GSDIa) and the company’s first approved gene therapy. The one-time intravenous AAV8 therapy…
