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Gene Therapy, 20 years in the making, produces remarkable results.

First Patient Receives Gene Therapy for GSD


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

The Phase 1/2 clinical trial, led by Dr. Weinstein in conjuction with the Pharmaceutical company Ultragenyx, based in Navato, California, has already administered the 2nd round of dosing with very positive results. Phase 3 trials will begin January 2022. 

Our foundation was instrumental in moving this ground breaking research forward by funding studies to develop the treatment and test its efficacy. We hope to continue this progress until the treatment becomes available for the GSD community. You can help by Donating Now.


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