Conditions / Genetic

metachromatic leukodystrophy

info · Genetic · ICD-10: E75.25

A sphingolipidosis characterized by the accumulation of sulfatides in cells, especially the myelin producing cells of the nervous system.

Signs and symptoms

  • Polyneuropathy
  • Hypotonia
  • Muscle weakness
  • Motor deterioration
  • Developmental regression
  • Global developmental delay
  • Peripheral demyelination
  • Peripheral neuropathy
  • Increased CSF protein concentration
  • Loss of ambulation

Medications that may treat it

atidarsagene autotemcel

Also known as: MLD; Scholz cerebral sclerosis; arylsulfatase A deficiency; deficiency of cerebroside-sulfatase; sulfatide lipoidosis