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- Trump nominates Heidi Overton as FDA Commissioner as agency releases CGT Q&A guidanceThe U.S. Food and Drug Administration issued final guidance that answers common questions about developing cellular and gene therapy products. The document aims to clarify regulatory expectations f…
- Ultragenyx secures FDA approval for Glenglycos, first gene therapy for GSD IaUltragenyx announced FDA approval of Glenglycos, a one-time gene therapy for glycogen storage disease type Ia, a rare genetic disorder. The therapy delivers a functional gene to the liver to resto…