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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 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…