Developing therapeutic approaches for metachromatic leukodystrophy

  title={Developing therapeutic approaches for metachromatic leukodystrophy},
  author={Shilpa A Patil and Gustavo H B Maegawa},
  booktitle={Drug design, development and therapy},
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 treating MLD is limited but expanding with a number of early stage clinical trials… CONTINUE READING
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