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GENGLYCOS approved and trading under the generic name pariglasgene brecaparvovec-opnr; shares rose after the news as the company awaits a second gene therapy decision.
People with Glycogen Storage Disease Type Ia can't safely go without food. Their bodies can't release stored sugar, so many eat raw cornstarch around the clock, including overnight, to keep blood sugar from crashing. On August 19, 2026, the FDA approved GENGLYCOS, the first treatment aimed at the genetic cause of the disease rather than its symptoms.
Updated Aug 20
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