Connecticut Children’s Medical Center and UConn Health will host the first gene therapy clinical trial for a rare genetic childhood disorder that affects the liver, the medical centers announced Thursday.
The U.S. Food and Drug Administration this week approved the trial for Glycogen Storage Disease (GSD), which will be tested in Connecticut under the direction of Dr. David Weinstein, a professor in the Department of Pediatrics at the UConn School of Medicine.
The local medical centers will collaborate with centers across the world in the clinical trials expected to begin this year.
Participants will focus on GSD, a rare genetic childhood disorder that affects the liver that impacts one of in every 100,000 people. The disease could be life threatening, officials said.
Weinstein said the gene therapy is the “next step” in finding a cure for the disease.
The GSD Program, a joint venture of Connecticut Children’s and UConn Health, is the world’s largest for care and treatment of the disease, which has drawn patients to Connecticut Children’s from 49 states and 48 countries.
