Gene therapy eases daily cornstarch burden for teen with rare metabolic disease
Thirteen-year-old Trey McGowan has a rare glycogen storage disease Type 1a (von Gierke) that requires uncooked cornstarch every few hours to prevent dangerous lows; after FDA-approved gene therapy (Genglycos) his daily cornstarch intake dropped and feeds are now six per day, easing the family’s anxiety and keeping him on a life-sustaining regimen that will always include cornstarch.