Disease specific therapies in leukodystrophies and leukoencephalopathies

Affiliations

• 1 Department of Neurology, Children's National Health System, Washington, DC, USA.
• 2 Department of Neurology, Lucile Packard Children's Hospital and Stanford University School of Medicine, Stanford, CA, USA.
• 3 Department of Pediatrics and Neurology, University of Utah School of Medicine, Salt Lake City, UT, USA.
• 4 Department of Pediatrics, Montreal Children's Hospital/McGill University Health Center, Montreal, Canada; Department of Neurology and Neurosurgery, Montreal Children's Hospital/McGill University Health Center, Montreal, Canada.
• 5 Department of Human Genetics and Pediatrics, McGill University and the Montreal Children's Hospital Research Institute, Montreal, Canada.
• 6 MLD Foundation, USA.
• 7 Department of Neurology, Mayo Clinic, Rochester, MN, USA; Department of Pediatrics and Medical Genetics, Mayo Clinic, Rochester, MN, USA.
• 8 The Moser Center for Leukodystrophies and Neurogenetics Service, The Kennedy Krieger Institute, Johns Hopkins School of Medicine, Baltimore, MD, USA.
• 9 The PMD Foundation, USA.
• 10 Department of Child Neurology, VU University Medical Center, and Neuroscience Campus Amsterdam, Amsterdam, The Netherlands.
• 11 Department of Pediatrics and Neurology, Oregon Health and Science University, Portland, OR, USA.
• 12 Mission Massimo Foundation Inc., Melbourne, VIC, Australia; Mission Massimo Foundation Inc., Los Angeles, CA, USA.
• 13 Center for Translational Neuromedicine and the Department of Neurology of the University of Rochester Medical Center, Rochester, NY, USA.
• 14 Center for Genetic Medicine Research, Children's National Health System, Washington, DC USA.
• 15 Department of Pediatrics, Papé Family Pediatric Research Institute, Oregon Health and Science University, Portland, OR, USA.
• 16 Department of Pediatrics, University of California, San Francisco, San Francisco, CA, USA; Department of Neurological Surgery, University of California, San Francisco, San Francisco, CA, USA.
• 17 Waisman Center and Department of Comparative Biosciences, University of Wisconsin-Madison, Madison, WI, USA.
• 18 Department of Neurology, Hunter James Kelly Research Institute-HJRKI, University of Buffalo School of Medicine and Biomedical Sciences, Buffalo, NY, USA; Department of Biochemistry, Hunter James Kelly Research Institute-HJRKI, University of Buffalo School of Medicine and Biomedical Sciences, Buffalo, NY, USA.
• 19 Institute of Metabolic Disease, Baylor Research Institute, Dallas, TX, USA.
• 20 Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.
• 21 Department of Pediatrics, University of Pittsburgh, One Children's Hospital Drive, Pittsburgh, PA, USA.
• 22 Department of Neurology, Children's National Health System, Washington, DC, USA; Center for Genetic Medicine Research, Children's National Health System, Washington, DC USA; Department of Integrated Systems Biology, George Washington University School of Medicine, Washington, DC, USA. Electronic address: avanderv@childrensnational.org.