MPS VI Clinical Surveillance Program
The purpose of this study is to characterize and describe the MPS VI population as a whole in a registry, including the differences, progression, and natural history of MPS VI. Participants in this observational study have been diagnosed with mucopolysaccharidosis VI (Maroteaux-Lamy Syndrome or MPS VI). MPS VI is a genetic disorder that results in the build up of sugar molecules called glycosaminoglycans (or GAGs) in body tissues and leads to a variety of physical problems. Physical problems may include among others, decreased growth, nose, throat and ear problems, heart disease, bowel problems and bone and joint problems. Other objectives are to help the MPS VI medical community with the development of recommendations for monitoring patients and reports on patient outcomes to optimize patient care and also to evaluate the long-term effectiveness and safety of Naglazyme.
Back to Clinical Trials list