CLEMSON UNIVERSITY

Clemson Researchers Test Existing Drugs for Sanfilippo Syndrome Using Fruit Fly Models

- Clemson University researchers are testing four FDA-approved drugs for potential treatment of Sanfilippo syndrome, a fatal neurodegenerative disease affecting children. - The study uses fruit fly models to evaluate drug efficacy, leveraging the fact that 70% of human disease genes have fruit fly counterparts. - The research focuses on addressing heparan sulfate buildup in cellular lysosomes, which causes progressive brain damage in affected children. - If successful in fruit flies, the drugs could advance to human clinical trials within the next few years at the Greenwood Genetic Center.