The University of Georgia Research Foundation, Inc. (UGA Research Foundation) received a $226,500 Project Grant from the National Institute of Neurological Disorders and Stroke (NINDS) under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853). The grant, awarded on February 1, 2025, aims to develop novel disease models for Mucopolysaccharidosis Type IIIA (MPS IIIA), also known as Sanfilippo Syndrome, and identify therapeutic agents to reduce the accumulation of heparan sulfate in MPS IIIA cells. Specifically, the project will utilize patient-derived induced pluripotent stem cells to generate peripheral neural stem cell models to study PNS phenotypes and dysfunction in MPS IIIA. Additionally, the project will repurpose FDA-approved drugs to reduce heparan sulfate biosynthesis as a potential substrate reduction therapy for MPS IIIA. The innovative strategies proposed aim to improve understanding of the molecular underpinnings of peripheral nervous system dysfunction in MPS IIIA and promote the discovery of new therapies.