Adeline Vanderver, MD Neurologist



Adeline Vanderver, MD, is the Director of the Myelin Disorders Clinic at Children’s National Health System. Dr. Vanderver is an international leader in the study of leukodystrophies, conditions that affect about one in 7,000 children.

Dr. Vanderver has been interested in solving the mysteries of inherited myelin disorders since she was a medical student. She was touched by families that had been trying for years to understand the medical condition their children were experiencing before receiving a diagnosis. Dr. Vanderver also is deeply involved in searching for genetic pathways that might explain how these conditions occur. Just as important, she says, is building community support for patients and their families.

The leukodystrophy research program has the following goals, all focused on the clinical and bench evaluation of subjects with leukodystrophy, or inherited disorders of the white matter of the brain:

1. Improved understanding of the mechanisms and natural history of Aicardi Goutières syndrome. AGS is caused by mutations in genes that encode nucleases and related proteins. It is hypothesized that accumulation of immunostimulatory DNA results in immune activation and destruction of neurologic tissue. We are the US site for a large international collaboration on AGS. We have run several patient clinics and have a biorepository. We are also currently exploring the role of autoantibodies in this disorder. Finally, we are starting a clinical trial in collaboration with the NIH (U01).

2. Improved understanding of the mechanisms of Vanishing White Matter disease. This disorder is caused by mutations in the cellular mechanisms for response to ER stress and initiation of protein translation (eIF2B or eukaryotic initiation factor 2B). We are exploring the effect of exaggerated endoplasmic reticulum stress on glial cells and propose a mechanism by which mutations in EIF2B would result in alteration in glial cell lineage development. This project uses existing primary glial cell cultures, a murine model and murine oligodendrocyte cell lines.

3. Improved understanding of the mechanisms of TUBB4A-related hypomyelination using glial cell models (Oli-neu) and neuronal models to assess the role of altered tubulin on trafficking and cellular integrity.

4. Undiagnosed Leukodystrophies Program. Over half of patients with inherited disorders of the white matter never achieve a specific diagnosis.  We have a consortium, the Myelin Disorders Bioregistry Project, with over 900 enrolled patients and family members, enrolling more than 100 new participants each year. We propose to test the effectiveness of Next Generation Sequencing (NGS) as a diagnostic tool for the leukodystrophies.

Dr. Vanderver is an Associate Professor in the Departments of Neurology and Pediatrics, and Integrative Systems Biology at George Washington University Medical Center. She also works with the National Human Genome Research Institute at the National Institutes of Health.

Education & Training

Education & Training

  • Fellowship Program, Child Neurology, 2004
    Children's National Medical Center
  • Residency Program, Pediatrics, 2001
    A.I. Dupont Hospital for Children
  • Internship Program, Transitional Internship, 1999
    Frankford Hospital of the City
  • MD, 1998
    Universite Catholique de Louvain
  • CEM, 1994
    Facultes universitaires Notre-Dame de la Paix
Patient Stories

Patient Stories: Adeline Vanderver

Massimo’s Story: Solving the Mystery of Leukodystrophy

Massimo's Story

During his first 11 months of life, Massimo was hitting all of his developmental milestones, and his parents, Stephen and Sally Damiani, didn’t notice anything out of the norm. Right before his first birthday, the family started noticing signs of regression in Massimo.

Emily Rose Wagner: Solving the Mystery of Leukodystrophy

Emily's Story

In 2012, Emily Rose Wagner was born in Boston, and her parents were beyond thrilled to have their second child. The first few months after Emily was born were a dream come true for the entire family.

Research & Publications

Research & Publications

35th Annual Meeting of the Child Neurology Society

(2006) Update on Leukodystrophies

57th Annual Meeting of the American Academy of Neurology

(2005) Cerebrospinal Fluid Proteome in Vanishing White Matter Disease/ Childhood Onset Ataxia and Central Hypomyelination: Decrease in asialotransferrin

58th Annual Meeting of the American Academy of Neurology

(2006) Decreased asialotransferrin in the cerebrospinal fluid in Vanishing White Matter disease/ Childhood Onset Ataxia and Central Hypomyelination: a clinical biomarker

American College of Medical Genetics Annual Clinical Genetics Meeting

(2008) Update on Leukodystrophies

BeckwithWiedemann syndrome

(2003) National Organization for Rare Disorders Guide to Rare Disorders.

Clinical and molecular phenotype of AicardiGoutières syndrome

(2007) American Journal of Human Genetics

Decreased asialotransferrin in cerebrospinal fluid of patients with Childhood Onset Ataxia and Central Nervous System Hypomyelination

(2005) Vanishing White Matter Disease

Genetic and Clinical Heterogeneity in eIF2Brelated disorder

(2008) Journal of Child Neurology

Giant Axonal Neuropathy

(2003) National Organization for Rare Disorders Guide to Rare Disorders

Turner syndrome

(2003) National Organization for Rare Disorders Guide to Rare Disorders

View publications on PubMed

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