Type 1 Diabetes: Closing in on a Cure

A UPMC Children’s physician-researcher and his team are closer than ever to discovering a new treatment for over 200,000 children, adolescents, and adults living with diabetes in the United States.

Innovation in Gene Therapy Starts at UPMC Children’s

UPMC Children’s is on its way to becoming the lead site for development and human clinical trials of a new gene therapy treatment for VLCADD (Very Long Chain Acyl CoA Dehydrogenase Deficiency), a rare genetic disorder that is identified through newborn screening.