Pariglasgene brecaparvovec for treating glycogen storage disease type 1a

Pariglasgene brecaparvovec is in development for the treatment of patients with glycogen storage disease type 1a (GSD1a). GSD1a is an inherited disorder caused by the lack of an enzyme (type of protein) called glucose-6-phosphatase (G6Pase) which aids in the breakdown of glycogen (a complex sugar stored in the body) to glucose (a simple sugar). This lack of efficient glycogen breakdown results in low blood sugar, and a build-up of glycogen in the liver, kidneys, and gut which can result in long-term complications such as liver and kidney disease and muscle weakness. Current treatment options for patients with GSD1a are limited and involve placing patients on strict uncooked cornstarch diets and the avoidance of fasting to prevent hypoglycaemia (low blood sugar). Current treatment does not correct the underlying cause of the disease, and patients remain at risk of serious long-term complications.