Tippi Mackenzie, MD was the Keynote Speaker on Wednesday, February 9, 2022. Tippi MacKenzie is a Professor of Surgery at the University of California, San Francisco and the Director of the Eli and Edythe Broad Institute for Regeneration Medicine. Read speaker bio.
Michael T. Osterholm, PhD, MPH was the Keynote Speaker on Wednesday, February 10, 2021 as well as Keynote Speaker Peter Marks, MD, PhD on Thursday, February 11, 2021. Read speaker bios.
Chester B. Whitley, PhD, MD delivered the keynote address on Wednesday, February 12, 2020 and Peter Marks, MD, PhD delivered the keynote address on Thursday, February 13, 2020. Dr. Whitley is the Principal Investigator for the Lysosomal Disease Network (LDN), a growing consortium of medical centers collaborating since 2003 and is the director of the Center for Biologics Evaluation and Research (CBER) at the U.S. Food and Drug Administration (FDA). Read speaker bios.
Chester B. Whitley, PhD, MD Delivered Keynote Address on Wednesday, February 12, 2019. Dr. Whitley is a tenured Professor in the Department of Pediatrics, the Department of Experimental and Clinical Pharmacology, and Director of the Advanced Therapies Program at the University of Minnesota. Read speaker bios.
Petra Kaufmann, MD, MSc delivered Keynote on Wednesday, February 7, 2018 and R. Rodney Howell, MD Delivered Keynote on Thursday, February 8, 2018. Petra Kaufmann is director of the NCATS Office of Rare Diseases Research. Her work includes overseeing the Rare Diseases Clinical Research Network program, Genetic and Rare Diseases Information Center, and the NCATS Toolkit for Patient-Focused Therapy Development. R. Rodney Howell, MD is Professor of Pediatrics and a Member of the Hussman Institute for Human Genomics, at the Miller School of Medicine of the University of Miami. Read speaker bios.
Elsa Shapiro, PhD, Delivered Keynote on Wednesday, February 15, 2017. Elsa Shapiro, PhD, is Professor of Pediatrics and Neurology in the Division of Pediatric Behavioral Neuroscience at the University of Minnesota. Read speaker bios.
Christopher P. Austin, MD, Delivered Keynote at WORLDSymposium 2016. Dr. Austin is director of the National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health (NIH). Read speaker bios.
Elcomed Laurie Muldowney, MD, (US FDA), Delivered Keynote at WORLDSymposium 2015 on Wednesday, February 11, 2015. Dr. Muldowney is Medical Officer for the Division of Gastroenterology and Inborn Error Products at CDER/FDA. Read speaker bio.