Browsing: Glycogen

Conditions

Pariglasgene Brecaparvovec-opnr Approved for Glycogen Storage Disease 1a

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According to a new announcement, the FDA has granted accelerated approval to pariglasgene brecaparvovec-opnr (Genglycos; Ultragenyx Pharmaceutical, Inc), a one-time, liver-directed AAV8 gene therapy, to reduce daily cornstarch intake as an adjunct to nutritional management in patients 8 years and older with glycogen storage disease type Ia (GSD1a).1 The approval, announced August 19, 2026, makes…