Steven Walkley, DVM, PhD, is Professor of Neuroscience, Pathology and Neurology at the Albert Einstein School of Medicine, Bronx, NY, where he is Director of the Rose F. Kennedy Intellectual and Developmental Disabilities Research Center. Dr. Walkley has been a leader in the field of lysosomal disease research and therapy for over 3 decades and has made major contributions to our understanding of the biology of NPC disease. His laboratory pioneered the use of substrate reduction therapy (i.e., miglustat) as a therapeutic approach for NPC disease, which is now widely used clinically and represents the only approved therapy for the disease. More recently, his lab discovered that HPBCD was efficacious in reducing lysosomal storage in NPC disease, and provided critical pre-clinical data to support the filing of an IND with the FDA, leading to the Phase 1/2a clinical trial. Dr. Walkley is a former Chair of the NNPDF SAB, and is a founding member of the Collaborative.
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