Clinical Research Studies

MPS IVA is a genetic disorder that results in the buildup of sugars called glycosaminoglycans (or GAGs) in your body tissues and leads to a variety of physical problems. These may include, among others, decreased growth; eye, throat, and ear problems; heart disease; lung problems; and bone and joint problems. Normally, there is a continuous building up and breaking down of GAGs in the body tissues. Special proteins called enzymes are needed to make this process happen. Individuals with MPS IVA are missing 1 specific enzyme called N-acetylgalactosamine-6-sulfatase (also called GALNS). Without this enzyme, GAG cannot be completely broken down and begins to build up. It is this build up of GAG that causes the physical problems. The goal of the Registry is to collect medical information on people with MPS IVA from around the world in an effort to better understand what happens to them over time and to help their doctors take care of them. The Registry doctor will also continue to monitor your safety if you are treated with Vimizim®.