VIMIZIM Website design
VIMIZIM (Elosulfase alfa) is a drug for the treatment of Morquio syndrome which is caused by a deficiency in the enzyme.
Morquio A is a rare inherited disease that affects major organ systems in the body. The disease is a form of mucopolysaccharidosis, which is a type of lysosomal storage disorder. People born with Morquio A can’t break down certain complex carbohydrates known as glycosaminoglycans (GAGs) because their bodies don’t make enough of an enzyme, or protein, called N-acetylgalactosamine-6 sulfatase (GALNS). This enzyme breaks down or recycles materials the body can’t use. When the body doesn’t produce enough of the enzyme, GAGs build up in tissues, bones, and major organs.