Mucopolysaccharidosis Type VI
One of the first steps after an initial diagnosis is to find a doctor who has experience treating Mucopolysaccharidosis Type VI (MPS VI). To find a doctor or center that specializes in metabolic genetic conditions or lysosomal storage disorders like MPS VI in the United States, you can use the Clinic Locator Tool provided by the Maroteaux-Lamy Syndrome Resource Center at http://www.maroteaux-lamy.com/en-us/pages/patient/ClinicLocator.aspx.
It is also important to decide if enzyme replacement therapy is a good option for you. The United States Food and Drug Administration (FDA) approved the use of a medication called galsulfase (also known as Naglazyme®) for the treatment of Mucopolysaccharidosis Type VI (MPS VI) in June of 2005. Galsulfase is an enzyme replacement therapy using a man-made version of arylsulfatase B, which is the enzyme that is not working properly in MPS VI. While galsulfase is not a cure for MPS VI, it helps reduce the amount of glycosaminoglycans building up in the body, which may help slow the disease process and improve symptoms. To learn more about galsulfase, talk to your doctor, visit www.naglazyme.com, or contact BioMarin Patient and Physician Support at 1-866-906-6100.
It may also be important for newly diagnosed individuals and their families to find a support group right away, as the emotional stress of the diagnosis can be significant. The National MPS Society has family support programs and educational resources.