galsulfase

Galsulfase is a recombinant form of the human enzyme N-acetylgalactosamine 4-sulfatase, also known as arylsulfatase B. It is used as an enzyme replacement therapy for patients with mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy syndrome), a rare inherited lysosomal storage disorder caused by deficiency of this enzyme. The absence or marked reduction in N-acetylgalactosamine 4-sulfatase leads to accumulation of glycosaminoglycans (GAG), particularly dermatan sulfate, resulting in progressive cellular and organ dysfunction. Galsulfase is administered intravenously and taken up into lysosomes via mannose-6-phosphate receptor-mediated endocytosis, where it catalyzes the cleavage of sulfate esters from terminal N-acetylgalactosamine 4-sulfate residues on GAGs, reducing their accumulation and improving clinical symptoms such as walking and stair-climbing ability[1][2][3][5][6].

Brand names

Naglazyme

Other names

recombinant human arylsulfatase Brecombinant human N-acetylgalactosamine 4-sulfatasearylsulfatase B