What happens in MPS VI?
A person with MPS VI lacks an enzyme the body needs to get rid of waste in the cells. This waste is a jelly-like product called GAG, which is short for “glycosaminoglycan” (gly-KOH-sah-meen-o-GLY-can).4
Over time, GAG builds up inside the cell making the cells swell. Packed with too much GAG, the cells don’t work right. Since body tissues and organs are made of the cells, tissues and organs don’t work right either. GAG is stored in most cells of the body, so many major organs and systems can be harmed. Most of the time intelligence is not affected.3,4
NAGLAZYME® (galsulfase) provides the enzyme that the body needs to break down and get rid of GAG. In timed tests, people who took NAGLAZYME were able to climb more stairs and walk farther than before.1
GAG-saturated cells distend and malfunction
On the left, a normal cell has just a few “lysosomes” (LIE-so-sohms). These are the clear areas that look like bubbles in the picture. Lysosomes store and break down the cell’s waste.
In MPS VI, the lysosomes don’t have an enzyme they need to break down a kind of waste material called GAG so that it can leave the cell. As GAG builds up, more and more lysosomes store GAG inside the cell. In the picture on the right you can see the result. The cell becomes swollen. It can’t work as well. This causes problems throughout the body.4
Next: What does MPS VI do?