[Harvard MIT CRS] Regulatory Science Forum

Towards Prevention of Genetic Prion Disease - A Patient-Scientist Driven Scientific and Regulatory Journey.

Sonia Vallabh, PhD co-runs a prion research lab at the Broad Institute, long with her husband, Eric Minikel. They both left their former careers to focus on biomedical research after Sonia had inherited a genetic mutation from her mother that led to a genetic prion disease - rapidly fatal and currently untreatable neurodegenerative disease that typically strikes in midlife. Sonia works on prion disease drug discovery, and focuses on lowering the amount of prion protein in the brain.