Pediatric Storage Disorders Lab

Leading the way in Batten disease research, the Pediatric Storage Disorders Lab (PSDL) spearheads global efforts to unravel the complexities of neuronal ceroid lipofuscinoses (NCL) and related lysosomal storage disorders. At the forefront of NCL studies and therapeutic innovations, our team combines advanced morphological analysis with cutting-edge approaches such as gene therapy, enzyme replacement and small molecule strategies. By mapping disease progression across diverse models, we aim to refine targeted interventions that offer hope for patients worldwide.