Naglazyme® (galsulfase) – PRODUCT INFORMATION

MPS VI (Mucopolysaccharidosis VI), also known as Maroteaux-Lamy syndrome, is an inherited lysosomal storage disorder caused by the deficiency of N-acetylgalactosamine 4-sulfatase (arylsulfatase B), an enzyme normally required for the breakdown of certain complex carbohydrates known as glycosaminoglycans (GAG). If the enzyme is not present in sufficient quantities, the normal breakdown of GAG is incomplete or blocked. The cell is unable to excrete the carbohydrate residues which then accumulate in the lysosomes of the cell. This accumulation disrupts the cell's normal functioning and gives rise to the physical manifestations of the disease.

MPS VI affects males and females equally, and in most cases, both parents of an affected child are asymptomatic carriers of the disease. MPS VI is a disease with a wide variation in the rate of disease progression, the severity of symptoms, and the organ systems affected. Unlike MPS I, MPS VI does not typically affect intelligence level.

Naglazyme is a trademark of BioMarin Pharmaceutical Inc.