Article
Developing therapeutic approaches for metachromatic leukodystrophy.
Drug design, development and therapy - 1 Jan 2013
Patil Shilpa A, Maegawa Gustavo H B
Abstract excerpt
Metachromatic leukodystrophy (MLD) is an autosomal recessive lysosomal disorder caused by the deficiency of arylsulfatase A (ASA), resulting in impaired degradation of sulfatide, an essential sphingolipid of myelin. The clinical manifestations of MLD are characterized by progressive demyelination and subsequent neurological symptoms resulting in severe debilitation. The availability of therapeutic options for...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.