Conditions We Treat

At the Stanford Medicine Children’s Health Pediatric Leukodystrophy Clinic, we specialize in the diagnosis and care of patients with known or suspected rare genetic disorders that affect the white matter of the brain. We aim to provide personalized treatment that meets the needs of our patients and their parents. Stanford Medicine Children’s Health is also home to an infusion center and some of the world’s foremost cutting-edge research and clinical trials.

Some of the leukodystrophies we treat:

  • Adrenoleukodystrophy (X-ALD)
  • Aicardi–Goutières syndrome
  • Alexander disease
  • Canavan disease
  • Krabbe disease (globoid cell leukodystrophy)
  • Hypomyelination with atrophy of the basal ganglia and cerebellum (H-ABC)
  • Leukoencephalopathy with brainstem and spinal cord involvement and lactate elevation (LBSL)
  • Megalencephalic leukoencephalopathy with subcortical cysts (MLC)
  • Metachromatic leukodystrophy (MLD)
  • Pelizaeus-Merzbacher disease (PMD)
  • Zellweger spectrum disorders (peroxisomal biogenesis disorders)
  • 4H syndrome (POLR3-related leukodystrophy)
  • Vanishing white matter disease (VWM)
  • Undiagnosed leukodystrophies