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Using translational approaches that encompass genomic studies, biomarker development, disease modeling, natural history studies, and clinical trials, Dr. Vanderver seeks to improve the quality of life and lifespan of individuals living with leukodystrophies or heritable disorder of myelin. She leads the Leukodystrophy Center of Excellence at Children's Hospital of Philadelphia.
Dr. Vanderver conducts translational research on leukodystrophies and leukoencephalopathies in order to refine clinical diagnostic tools and accelerate the development of therapeutic treatments.
Along with her team of researchers, Dr. Vanderver aims to define novel homogeneous groups of patients with previously unclassified leukodystrophies and to uncover the genetic causes of these disorders; establish the molecular disease mechanisms in selected known leukodystrophies; assess the validity of advanced genetic sequencing techniques in the diagnosis of these disorders; and develop the next generation of therapeutic clinical trials through natural history and biomarker discovery studies.
She is also Program Director of CHOP's Leukodystrophy Center of Excellence (LCE), which is focused on creating new standards of care for children with leukodystrophies by advancing leukodystrophy gene discovery, creating new therapies, and supporting and advocating for patients and their families.
In parallel with this strong clinical program, Dr. Vanderver's preclinical and clinical research projects aim to discover molecular therapeutics that target the genetics of leukodystrophy subtypes. In addition to her clinical and research efforts, Dr. Vanderver leads the Global Leukodystrophy Initiative, an advocacy group that includes parents, clinicians, and researchers, to raise disease awareness and ensure that patients receive appropriate social and medical support.
Education and Training
Candidature en Médecine, Facultés Universitaires Notre Dame de la Paix Namur, Belgium 1991-1994
Medical Doctorate, Universite Catholique de Louvain Brussels, Belgium, 1994-1998
Transitional Year Residency, Frankford Hospital, Temple University, 1998-1999
Pediatric Residency (PL-1 and PL-2), A.I. duPont Hospital for Children, Thomas Jefferson University, 1999-2001
Child Neurology Fellowship, Children’s National Medical Center, 2001-2004
Biochemical Genetics Fellowship, National Human Genome Research Institute/National Institutes of Health, 2006-200
Titles and Academic Titles
Program Director, Leukodystrophy Center of Excellence
Jacob A. Kamens Endowed Chair in Neurologic Disorders and Translational Neurotherapeutics
Associate Professor of Neurology, Children's National Health System, George Washington University School of Medicine and Health Sciences
Fellowship Selection Committee - Department of Neurology, Children’s National, 2005-2016
Individualized Professional Agreement and Special Volunteer status - National Human Genome Research Institute/National Institutes of Health - Principal Investigator of “Leukodystrophies of Unknown Cause”, 2008-2016
Safety Initiative - Department of Neurology - assessed safety of reporting of lab results for the Department of Neurology, Children’s National, 2009-2010
Biochemical Laboratory - reading of biochemical laboratory tests, Children’s National, 2008-2012
Pediatric Residency Selection Committee- Department of Pediatrics, Children’s National, 2009-2011
Consultation in the Undiagnosed Disease Program - National Human Genome Research Institute/National Institutes of Health, 2009-2016
Scientific Advisory Board – European Leukodystrophy Association, 2016-
Medical and Scientific Advisory Board – United Leukodystrophy Foundation, 2017-
Journal Reviewer - ad hoc reviews in the field of leukodystrophy for Neurology, Journal of Pediatric Neurology, Clinical Autonomic Research, Pediatric Neurology, Orphanet, Brain, American Journal of Human Genetics and others, Ongoing
Cellular and Molecular Biology of Glia Study Section at the Center for Scientific Review, National Institutes of Health, 2018-
Outstanding Junior Member Award of the Child Neurology Society, 2003
Early Career Reviewer (ECR) Program at the Center for Scientific Review (CSR), National Institutes of Health, 2013
Murdoch Children's Research Institute Visiting Fellows Award, Royal Children's Hospital of Melbourne, Australia, 2015