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- 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…
- Regeneron wins FDA approval for Pasatru, targeting rare bone disease FOPThe U.S. Food and Drug Administration has approved Regeneron Pharmaceuticals' drug Pasatru for the treatment of fibrodysplasia ossificans progressiva (FOP), a very rare condition in which muscle an…