A medication used to treat a rare genetic disorder called Mucopolysaccharidosis type VI or Maroteaux-Lamy syndrome, which causes the body organs to be enlarged and inflamed.


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.


Galsufase is a variant form of the polymorphic human enzyme N-acetylgalactosamine 4-sulfatase of recombinant DNA origin. Galsulfase is a glycoprotein with a molecular weight of approximately 56 kD. The recombinant protein is comprised of 495 amino acids and contains six asparagine-linked glycosylation sites, four of which carry a bis mannose-6-phosphate manose7 oligosaccharide for specific cellular recognition. Post-translational modification of Cys53 produces the catalytic amino acid residue Ca-formylglycine, which is required for enzyme activity and is conserved in all members of the sulfatase enzyme family.



For the treatment of adults and children with Mucopolysaccharidosis VI.


Mucopolysaccharide storage disorders are caused by the deficiency of specific lysosomal enzymes required for the catabolism of GAG. Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy syndrome) is characterized by the absence or marked reduction in N-acetylgalactosamine 4-sulfatase. The sulfatase activ... Read more

Mechanism of action

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-su... Read more


Information currently not available.

Protein binding

Information currently not available.

Volume of distribution

Week 1: 56-323 mL/kg and 59-2799 mL/kg by week 24


Information currently not available.

Half life

9 (6 to 21) minutes during the first week of treatment, 26 (8 to 40) minutes by the 24th week.

Route of elimination

Information currently not available.


There is no experience with overdose of galsulfase.

Adverse Effects


Information currently not available.

Food Interactions

    Information currently not available.


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