Active Ingredient(s):Vestronidase alfa-vjbk FDA Approved: * November 15, 2017 Pharm Company: *ULTRAGENYX PHARM INC Category:Genetic Disorders
Sly syndrome, also called mucopolysaccharidosis type VII (MPS-VII), is an autosomal recessive lysosomal storage disease caused by a deficiency of the enzyme β-glucuronidase. This enzyme is responsible for breaking down large sugar molecules called glycosaminoglycans (AKA GAGs, or mucopolysaccharides). The inability to break down GAGs leads to a buildup in many tissues and organs of the body. The severity of the disease can vary widely.
1 Signs and symptoms
* May have multiple approval dates, manufacturers, or labelers.