Leukodystrophy and Myelin Disorders Program

The Leukodystrophy and Myelin Disorders Program at Children's National Hospital is a recognized leader in providing comprehensive clinical care, diagnostic testing and the most advanced treatments for children with inherited white matter diseases, or leukodystrophies.

Choosing the Leukodystrophy and Myelin Disorders Program at Children’s National

Identifying the cause and type of a white matter disease can be challenging. To ensure that your child receives the best care and the most appropriate form of treatment, the Leukodystrophy and Myelin Disorders Program provides your child with integrated care from a team of neurologists, biochemical geneticists, pediatricians, rehabilitation physicians, genetic counselors and speech therapists. 

Features of our program include:

• Expert care. Our specialists are international leaders who have pioneered leukodystrophy and myelin disorder research and treatments.
• Personalized treatment plan. We create a personalized care plan based on the genetic makeup of your child. We understand that all children are unique and require treatments that meet their unique needs.
• Clinical trials and research. Our team of researchers and clinicians are devoted to the discovery of novel variations responsible for pediatric white matter disorders and to translate these findings into clinical treatments through extensive research, clinical trials and studies.
• Ongoing care. Our team of specialists provides ongoing care throughout the diagnosis and treatment of your child’s condition.

Conditions we treat include:

• Aicardi Goutières Syndrome
• Adrenoleukodystrophy, Adrenomyeloneuropathy
• Alexander Disease
• Canavan Disease
• Cockayne Syndrome
• Fucosidosis
• Hypomyelination with Atrophy of Basal ganglia and Cerebellum (H-ABC)
• Krabbe disease
• Leukoencephalopathy with Calcifications and Cysts (Labrune Syndrome)
• Metachromatic leukodystrophy
• Megalencephalic Leukoencephalopathy with Subcortical Cysts (MLC)
• Pelizaeus Merzbacher Disease, Pelizaeus Merzbacher-like disease
• PolR3 related disorders (4-H syndrome)
• tRNA synthetase disorders (LTBL/EARS2), LBSL/DARS2, FARS2, others)
• Vanishing White Matter disease
• Zellweger spectrum disorders