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- FDA approves Ultragenyx gene therapy for glycogen storage disorder; Overton faces confirmation hurdlesThe U.S. Food and Drug Administration has granted approval to Ultragenyx's gene therapy aimed at treating a glycogen storage disorder. This marks a regulatory milestone for the company and the rare…
- 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…