- 17 Downloads
Galsulfase [Aryplase™, arylsulfatase B, BM 102, Naglazyme™, rhASB, recombinant human N-acetylgalactosamine-4-sulfatase, recombinant human arylsulfatase B] is under development with BioMarin Pharmaceutical as an enzyme replacement therapy for the treatment of mucopolysaccharidosis (MPS) VI.
MPS VI (also known as Maroteaux-Lamy syndrome) is a progressive, debilitating genetic disease resulting in early death. Patients with MPS VI have a deficiency in the arylsulfatase B (ASB) enzyme that is essential for the progressive breakdown of certain complex carbohydrates. The deficiency in ASB results in the build-up of carbohydrate residues in the lysosomes in all cells of the body. Patients are usually diagnosed at 6–24 months of age, and the symptoms include deceleration of growth, enlarged liver and spleen, skeletal and joint deformities, and upper airway obstruction. Patients do not survive past 20–30 years of age in the more severe cases, but may live longer with the milder cases,...
KeywordsEnzyme Replacement Therapy Arylsulfatase Orphan Drug Status Biologics License Application Galsulfase
- 1.BioMarin Pharmaceutical Inc. BioMarin Submits U.S. Biologics License Application for Marketing Authorization of Aryplase for MPS VI. Media Release: 29 Nov 2004. Available from URL: http://www.bmrn.com