In July 2018 gene therapy for the treatment of GSD became a reality when the first dose was administered to a human patient at Uconn John Dempsey Hospital. The gene therapy was was given to Jerrod Watts (GSD1a) through a one time 30-minute IV infusion by Dr. David Weinstein, the former Director of the Glycogen Storage Disease Program at the Connecticut Children's Medical Center, and his team at UConn Health.

The gene therapy is carried through the patient's bloodstream by a naturally occurring virus to the liver to replace deficient enzymes causing the disease and to jumpstart the body's glucose control. Three patients received the lowest dose of treatment and initial results are very positive. All three patients demonstrated improvement in glucose control throughout the day and all have been able to significantly decrease their daily cornstarch intake.  

Find out how that patient is doing today