There is currently no cure for Glycogen Storage Disease (GSD). The only approved treatment available involves the precise, continuous administration of cornstarch. If one dose is missed, the consequences can be fatal.
Dr. Weinstein’s research lab, located at UConn Health, focuses on ways to prevent the complications of the disease—and on discovering a cure.
His team is on the verge of clinical trials for the first gene therapy trial for GSD Ia, developed in conjunction with Dimension Therapeutics of Cambridge, MA. Testing of the gene therapy has shown great effectiveness in improving the health and life expectancy of canines born naturally with the disease.