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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 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…