Amsterdam UMC, the Netherlands

Marjo van der Knaap is an adult and pediatric neurologist, employed at Amsterdam UMC since 1991. She is head of pediatric neurology. In 2000 she founded the Amsterdam Leukodystrophy Center, where patient care is combined with fundamental, translational, and clinical research. She contributed to the MRI definition of VWM. In 2001/2002 her group discovered 5 VWM-related genes. Her research contributed to insight into VWM pathophysiology and therapy targets. She initiated the first therapy trial in VWM.

Primary Children’s Center for Pediatric Personalized Medicine, USA

Josh Bonkowsky is a pediatric neurologist specializing in clinical care and research. He is a professor of Pediatrics at the University of Utah Eccles School of Medicine. He is the Division Chief of Pediatric Neurology at Primary Children’s Hospital and the Director of the Primary Children’s Center for Personalized Medicine.

Principal Investigator

Prof. Orna Elroy-Stein is a Full professor in the Department of Cell Research and Immunology at the Faculty of Life Sciences, Tel Aviv University, and a member of the Sagol School of Neuroscience. She received her B.Sc. and M.Sc. from the Technion, her Ph.D. on Human Molecular Genetics at the Weizmann Institute of Science, Israel, and her postdoctoral training at the Lab of Viral Diseases, NIAID, Maryland, USA.

The Bradbury Laboratory is dedicated to understanding disease mechanisms and developing therapeutic approaches for rare, pediatric neurodegenerative disorders. A primary focus of the Bradbury lab is improving AAV targeting of myelinating cells, a current limitation of AAV, in order to advance therapeutic outcomes for leukodystrophies and other white matter disorders. The Bradbury lab is translational in nature with a commitment to safely and efficiently moving therapies into the clinic.