Treating an MPS VI patient with NAGLAZYME® (galsulfase) Patient compliance In order for the patient to benefit from treatment, regular ERT is necessary. If therapy stops, GAG builds up and symptoms may return. Patients and families may have unrealistic expectations about treatment outcomes. It is important to help patients and families understand that results vary and some improvements occur over a long period of time. Efficacy of ERT for MPS VI seen in the clinical trial was measured over a 6-month period. Long-term compliance requires doctor-patient coordination MPS VI patients may experience exceptional difficulties that can interfere with long-term compliance. It is important that these difficulties be addressed promptly and proactively. MPS VI patients may have difficulty making infusion appointments due to work and school obligations, or frequent illness or hospitalization. Be as flexible as possible in scheduling or rescheduling appointments Painful IV insertion can be a cause for anxiety which could reduce compliance. Consider the use of topical anesthetics to reduce the pain of IV insertion Transportation and other problems may arise. Utilize social services or contact your BioMarin Clinical Sales Specialist for advice Palliative care as individual symptoms present Palliative treatment can address the individual complications of MPS VI as they present. Palliative care often includes antibiotics, pain management, and surgery. Disease-specific therapies, such as enzyme replacement therapy (ERT), target the deficiency of enzyme activity and may help alleviate some symptoms. Due to the multisystemic nature of MPS VI, treatment requires a multidisciplinary approach.4 Surgery is common in MPS VI patients, with each procedure carrying specific risks of morbidity and mortality, and specific expected gains for the patient. MPS VI patients typically undergo multiple surgeries during their lifetime. Because of cardiac and respiratory comorbidities, special caution should be exercised when administering general anesthesia. Psychosocial support Psychosocial support is often a necessary part of a treatment plan for patients with MPS VI. Counseling and interaction with other patients and caregivers in support groups can help families cope with the impact of the disease.4,12 For a list of patient advocacy and support groups click here.