About MPS VI
MPS VI (or Maroteaux‐Lamy syndrome) is a rare but serious genetic disease. Only about 1100 people in the world are thought to have it. People born with MPS VI tend to have a cluster of symptoms that may include:
- Short stature
- Coarse facial features
- Stiff joints
- Breathing problems
- Difficulty walking/hip pain
A lysosomal storage disorder
The name “Maroteaux-Lamy” comes from the doctors who first described the disease in 1963. The other name of the disease, MPS VI, stands for the medical name of the disease, mucopolysaccharidosis VI.
MPS VI is a kind of disease called a “lysosomal storage disorder” or LSD. There are more than 40 known LSDs. In all of these diseases, the lack of specific enzymes causes waste to build up inside cells.
Inherited—and often hard to diagnose
MPS VI is an inherited condition. A child who gets one MPS VI gene from each parent will have the disease. There are different degrees of MPS VI. Some people have clear symptoms very early that grow worse quickly. Doctors call this “rapidly progressing” MPS VI. Some people may not show signs until later and have symptoms that get worse more slowly. This is called “slowly progressing” MPS VI. Regardless of whether a person has “rapidly” or “slowly” progressing MPS VI, the disease gets worse over time in all cases. This is why it is important to begin therapy as early as possible.
A treatment for MPS VI
NAGLAZYME® (galsulfase) replaces the natural enzyme that is missing in MPS VI. In timed tests, people who took NAGLAZYME were able to climb more stairs and walk farther than before.Next Page