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Hypomyelination with atrophy of basal ganglia and cerebellum

ORPHA:139441· ICD-10 E75.2

Definition

A rare disorder characterized by slowly progressive spasticity, extrapyramidal movement disorders (dystonia, choreoathetosis and rigidity), cerebellar ataxia, moderate to severe cognitive deficit, and anarthria/dysarthria. Patients present with distinctive MRI findings of diffuse hypomyelination of the cerebral hemispheres, mild to severe cerebellar atrophy, and atrophy of the basal nuclei.

Prevalence
<1 / 1 000 000
Inheritance
Autosomal dominant, Autosomal recessive
Age of onset
Childhood, Infancy, Neonatal