WORLDSymposium 2018 Keynote Speakers

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.

WORLDSymposium 2018 Patient Advocate Leader (PAL) Award

WORLDSymposium 2018 Patient Advocate Leader (PAL) Award was received by Jack Johnson on Wednesday, February 7, 2018. Jack is a founding member and Executive Director of the Fabry Support & Information Group (FSIG) in the United States, and is also a founding member and Vice President and Board Member of the Fabry International Network (FIN). More details.

WORLDSymposium 2018 Roscoe O. Brady Award

Mark E. Haskins, VMD, PHD Received the 2018 Roscoe O. Brady Award for Innovation and Accomplishment on Tuesday, February 6, 2018. Dr. Haskins has a career that spans over 40 years, from his beginning as a NIH Medical Genetics Trainee in 1975 working on MPS VI, through his appointment as Professor Emeritus in 2014. More details.

WORLDSymposium 2018 Program

Following the presentation of the Award for Innovation and Accomplishment in lysosomal disease research, presentations in the morning and afternoon sessions 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.