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- Regeneron’s ultra-rare bone disorder drug receives U.S. approval after 30-year developmentRegeneron Pharmaceuticals announced that its therapy for an ultra-rare condition in which bone forms in soft tissue has been approved by the U.S. Food and Drug Administration. The disease, sometim…
- 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…