Metachromatic leukodystrophy - LEUKOTREAT, therapeutic strategies for leukodystrophy affected patients

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Last update : 09/02/2015

Metachromatic leukodystrophy

Metachromatic leukodystrophy (MLD) is a neurodegenerative disease caused by deficiency of a lysosomal enzyme called arylsulfatase A.


Onset can occur during childhood, adolescence or in adulthood and is characterized by rapid demyelination of the central and peripheral nervous system associated with sulfatide accumulation in the brain and kidneys.

It is estimated that this disease affects 1/45 000 children at birth.

For detailed information regarding the different forms of MLD, its genetics, pathophysiology, diagnosis, genetic counseling, treatment options, visit ELA page on MLD.