MedPath

Galsulfase

Generic Name
Galsulfase
Brand Names
Naglazyme
Drug Type
Biotech
Chemical Formula
-
CAS Number
552858-79-4
Unique Ingredient Identifier
59UA429E5G
Background

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.

Indication

For the treatment of adults and children with Mucopolysaccharidosis VI.

Associated Conditions
Mucopolysaccharidosis Type VI
Associated Therapies
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globenewswire.com
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Global Mucopolysaccharidosis Treatment Market to Witness Growth

The global mucopolysaccharidosis treatment market, valued at USD 2,669.1 million in 2024, is projected to grow at a CAGR of 5.9% to USD 5,014.5 million by 2035. Growth is driven by advancements in biotechnology, enhanced diagnostic methods, increased awareness, and the availability of enzyme replacement therapy. Clinical trials for innovative treatments are expanding, despite high costs and lack of reimbursement policies limiting market growth.
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Global Mucopolysaccharidosis Treatment Market to Witness Robust Growth, Projected to Reach USD 5,014.5 Million by 2035 Growing at a CAGR of 5.9%

The global mucopolysaccharidosis (MPS) treatment market, valued at USD 2,669.1 million in 2024, is projected to grow at a CAGR of 5.9% to USD 5,014.5 million by 2035. Growth drivers include advancements in biotechnology, enhanced diagnostic methods, increased awareness of rare diseases, and the availability of enzyme replacement therapy (ERT). Clinical trials and new drug approvals, such as Lysogene's LYS-SAF302 and JCR Pharmaceuticals' JR-141, are pivotal. Despite high treatment costs and reimbursement challenges, the market is set to expand, with ERT remaining the preferred treatment.
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