The Food and Drug Administration has approved the first treatment for glycogen storage disease type Ia, a rare inherited disorder in which the liver cannot release stored sugar and blood glucose can fall to dangerous levels between meals. The agency granted accelerated approval to Genglycos, also called pariglasgene brecaparvovec-opnr, a one-time gene therapy from Ultragenyx for adults and children 8 and older.
For the 1,500 to 2,500 people the company estimates live with the condition in the United States, the practical stake is not abstract. Management means swallowing measured doses of raw cornstarch around the clock, including through the night, to keep blood sugar from crashing. Families set alarms. Teenagers wake up to eat. A missed dose can mean a seizure or a hospital visit.
