Other names: Morquio Syndrome; MPS IV
Mucopolysaccharidosis Type IV is a rare, lysosomal storage disease caused by a deficiency in N-acetylgalactosamine-6-sulfate sulfatase (GALNS) which is an enzyme involved in glycosaminoglycan (GAG) metabolism. Excessive storage of glycosaminoglycans can lead to problems with bone development, growth and mobility.
Drugs used to treat Mucopolysaccharidosis Type IV
| Name | Drug Class |
|---|---|
| Elosulfase alfa | Lysosomal enzymes |
| Vimizim | Lysosomal enzymes |
