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- 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…
- FDA approves Regeneron’s garetosmab for ultra-rare bone-forming diseaseThe FDA has granted approval to Regeneron's antibody garetosmab, which targets activin A, for the treatment of fibrodysplasia ossificans progressiva (FOP). FOP is an ultra‑rare disorder that causes…