FDA Grants Accelerated Approval to Ultragenyx’s Genglycos Gene Therapy
▫️Ultragenyx Pharmaceutical Has Received Accelerated Approval From The US Food And Drug Administration (FDA) For Its Gene Therapy, Genglycos (Pariglasgene Brecaparvovec-opnr), to Treat Glycogen Storage Disease Type Ia (GSDIa).
▫️The Approval Covers Both Adult And Paediatric Patients Aged Eight Years And Above.
▫️GSDIa Is A Rare Genetic Metabolic Disorder Caused by A Deficiency in The Enzyme Required For Releasing Glucose From The Liver Into The Bloodstream.
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