Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome

Study of Administration of Intravenous Naglazyme® Following Allogeneic Transplantation for Maroteaux-Lamy Syndrome

This is a single center study in which Naglazyme® will be given weekly for two years in patients with Maroteaux-Lamy syndrome, also known as mucopolysaccharide VI (MPS VI), who have previously been treated with an allogeneic transplant.

Inclusion Criteria:

• Mucopolysaccharidosis type VI (MPS VI, Maroteaux-Lamy syndrome) treated with a prior allogeneic transplant >2 years previously
• Persons currently receiving Naglazyme may be accepted into the study
• Age > 2 years
• >10% engrafted based on most recent testing
• Willing to commit to traveling to the University of Minnesota every 6 months
• Written informed consent with parent/guardian consent for children < 18 years of age or persons unable to consent with minor assent if appropriate

Exclusion Criteria:

• History of cardiac or pulmonary insufficiency or those requiring continuous supplemental oxygen
• Pregnant or breastfeeding
• Any condition that, in the view of the investigator, places the patient at high risk of poor treatment compliance or of not completing the study