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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…
- FDA grants approval to Ultragenyx's Genglycos gene therapy for ultra-rare glycogen storage disorderUltragenyx announced that the U.S. Food and Drug Administration has approved its gene therapy called Genglycos. The therapy targets an ultra-rare glycogen storage disorder that can lead to severe…