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.
WORLDSymposium 2020 Patient Advocate Leader (PAL) Award was received by Cara O’Neill, MD on Wednesday, February 12, 2020. Cara O’Neill, MD, is the Chief Science Officer at Cure Sanfilippo Foundation, a pediatrician, and mother to a daughter with the rare disease Sanfilippo syndrome (MPS III). More details.
WORLDSymposium 2020 Roscoe O. Brady Award was received by John F. Crowley, MD for Innovation and Accomplishment on Tuesday, February 11, 2020. John F. Crowley is the Chairman and CEO of Amicus Therapeutics, a global biotechnology company focusing on developing treatments for rare genetic diseases. More details.
Following Emerging Trends in the morning and the Young Investigator Awards, presentations in the afternoon session discussed innovations in technology and how they can be applied to early diagnosis for lysosomal conditions, progress in gene therapy, and exploitation of differences at the cellular level that may indicate early disease state. View full program.