Summary of Clinical Use

Approved to treat mucopolysaccharidosis type IVA (Morquio A syndrome), a lysosomal storage disease characterised by an inability to break down keratin sulphate which causes spondylo-epiphyso-metaphyseal dysplasia and results in musculoskeletal abnormalities and growth arrest. Sufferers may also experience nervous complications, respiratory problems, hepatomegaly, valvulopathies, hearing loss and corneal clouding.

Mechanism Of Action and Pharmacodynamic Effects

This drug replaces the enzyme deficient in Morquio A syndrome patients, restoring lysosomal catabolism of glycosaminoglycans such as keratin sulphate [1].