World’s first gene therapy for glycogen storage disease produces remarkable results

The rare and deadly genetic liver disorder, GSD type Ia, affects children from infancy through adulthood, causing dangerously low blood sugar levels and constant dependence on glucose consumption in the form of cornstarch every few hours for survival. If a cornstarch dose is missed, the disease can lead to seizures and even death. A clinical trial originally set out to simply test the safety and dosage of the gene therapy for three patients with GSD Type Ia. The dramatic improvement in their lives was unexpected.