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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 first gene therapy approval for GSDIa as Sanfilippo asset decision pendingUltragenyx received accelerated FDA approval for its gene therapy DTX401, marketed as Genglycos, to treat glycogen storage disease type Ia (GSDIa). The approval comes as the company awaits a secon…