Rare Diseases

Presentation by Susan Hayflick & Penny Hogarth. We know the cause of a devastating childhood disease. While we can’t yet correct the gene mutation that causes this disease, we have found a way to restore normal function. Moving this promising treatment from bench to bedside has involved fighting the evils of drug company greed. Susan Hayflick, M.D. is Chair of the Department of Molecular & Medical Genetics at OHSU. Penny Hogarth, M.D., is an associate professor of Genetics and Neurology at OHSU.