Vanishing White Matter Research

Marjo van der Knaap, MD, PhD

 

​Pediatric neurologist, VU University Amsterdam

As one of the first to identify the cause of VWM, Dr van der Knaap is at the forefront of VWM research.  Her research team continues to make good progress towards understanding VWM and how we may be able to treat it.  She is also the leading expert in identifying Leukodystrophies.

Orna Elroy-Stein, PhD

​Professor, Tel Aviv University

Israel

Dr. Elroy-Stein and her team were the first to develop a "mouse model" with the VWM gene. They are also making good progress understanding VWM, and are working to identify potential drug treatments for VWM.

Joshua Bonkowsky, MD, PhD

​Pediatric Neurologist, University of Utah

Salt Lake City, USA

The Bonkowsky lab is using a novel small vertebrate model (zebrafish) to understand VWM and to discover new treatments. Zebrafish offer key benefits to circumvent research issues: myelin development occurs rapidly; the same genes control myelin development in zebrafish as in humans; and low costs and small size facilitate screening not possible in other systems. Because the VWM zebrafish rapidly develop disease, we are using them to screen for drugs and compounds that help prevent the disease.  

© 2019 Vanishing White Matter Foundation