recombinant human arylsulfatase A

Recombinant human arylsulfatase A (rhARSA) is a protein-based enzyme replacement therapy designed to substitute for deficient or defective endogenous arylsulfatase A. Arylsulfatase A is an enzyme responsible for the degradation of sulfatides such as cerebroside 3-sulfate into cerebroside and sulfate. Deficiency of this enzyme leads to metachromatic leukodystrophy (MLD), a rare lysosomal storage disorder characterized by progressive demyelination in the central and peripheral nervous systems. The recombinant form (rhARSA) is produced using biotechnology methods and may be engineered as a fusion protein with additional domains to enhance delivery or targeting—for example, fused with an antibody fragment targeting transferrin receptors to facilitate blood-brain barrier crossing[2][6]. Its primary mechanism is direct enzymatic replacement of missing or dysfunctional ARSA activity in patients with MLD.

Other names

recombinant human arylsulfatase Arecombinant arylsulfatase AARSAASA