Loren DM Pena, MD, PhD

Biography

Loren Pena, MD, PhD, has served as principal investigator for multiple clinical trials focused on rare disorders and spanning different treatment approaches that include gene therapy. She has also described several new genes involved in rare disorders. The goal of her clinical research is to facilitate development of new therapeutic approaches and to assess the safety and efficacy of candidate treatments for rare and orphan diseases via longitudinal observation/natural history and interventional studies.