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- 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…
- FDA grants accelerated approval to Genglycos, first therapy for glycogen storage disease type IaThe U.S. Food and Drug Administration issued an accelerated approval for Genglycos (pariglasgene brecaparvovec-opnr), marking the first therapy for patients aged eight years and older with glycogen…