Welcome to the Florida Institute for Pediatric Rare Diseases at the Florida State University College of Medicine, a ground-breaking program of the FSU Health initiative. Our mission is to transform the lives of children affected by rare diseases through research, education, diagnosis, and clinical care.
Congenital hyperinsulinemia: IPRD-supported researcher Julia Wang, Ph.D., Assistant Professor of Biomedical Sciences at the Florida State University College of Medicine, is investigating congenital hyperinsulinism, a rare pediatric disorder characterized by excessive insulin secretion. This condition can cause dangerously low blood sugar levels, leading to seizures, brain injury, or even death. Current treatment options are limited, often relying on trial-and-error medication regimens and, in severe cases, surgical removal of pancreatic tissue.
Wang’s laboratory aims to transform this approach by applying functional genomics and CRISPR-based tools to connect specific genetic mutations with individual treatment responses. With support from research assistants Carson Mickey, Pilar Toledo, and Pamela Sandoval Sanchez, the team seeks to identify effective, tailored therapies for each patient. IPRD Director Pradeep Bhide noted that this research paves the way toward personalized precision medicine and improved outcomes for children affected by this devastating disorder.
