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Galsulfase is a recombinant human enzyme used as replacement enzyme therapy for the treatment of of adults and children with Mucopolysaccharidosis VI, a rare genetic disorder caused by a deficiency of a lysosomal enzyme.
Galsulfase is a recombinant human enzyme used as replacement enzyme therapy for the treatment of of adults and children with Mucopolysaccharidosis VI, a rare genetic disorder caused by a deficiency of a lysosomal enzyme.
Galsulfase supplies recombinant-engineered galsulfase, a normal variant form of the polymorphic human enzyme, N-acetylgalactosamine 4-sulfatase. It is a lysosomal hydrolase that catalyzes the cleavage of the sulfate ester from terminal N-acetylgalactosamine 4-sulfate residues of GAG chondroitin 4-sulfate and dermatan sulfate. Increased catabolism of GAG in turn reduces systemic dermatan sulfate accumulation, thereby reducing the primary symptoms of MPS VI.
For the treatment of adults and children with Mucopolysaccharidosis VI.
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