Metachromatic Leukodystrophy Medication

Updated: Jan 22, 2021
  • Author: Anna V Blenda, PhD; Chief Editor: Luis O Rohena, MD, PhD, FAAP, FACMG  more...
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Medication Summary

Drug therapy is not a component of the standard of care for metachromatic leukodystrophy|. Supportive care is provided for complications or symptomatic relief. Recombinant human arylsulfatase A (rhARSA) enzyme is available in Europe and has been designated as an orphan drug in the United States.