Treatments ERT

MPS I

Aldurazyme, administered once-weekly, is approved in over 60 countries worldwide, including the United States and European Union for long-term enzyme replacement therapy in patients with a confirmed diagnosis of MPS I, to treat the non-neurological manifestations of the disease. Aldurazyme was developed by BioMarin and Genzyme under a joint venture agreement that assigns commercial manufacturing responsibilities to BioMarin, and worldwide sales and marketing responsibilities to Genzyme.

Additional information can be obtained at www.aldurazyme.com or by contacting Genzyme at 800-745-4447.

MPS II

Elaprase is a long term enzyme replacement therapy for patients with a confirmed diagnosis of MPS II which has been approved for use in the US, Canada, and many countries in Europe. Elaprase was developed and is produced by Shire Human Genetic Therapies (formerly TKT), and is given as weekly infusions to replace the missing enzyme that Hunter syndrome patients fail to produce in sufficient quantities.

Additional information can be obtained at www.shire.com or by contacting OnePathSM
toll free 866-888-0660. OnePath provides assistance with insurance, product access, treatment centers and education about Elaprase and MPS II.

MPS IVA

Vimizim™ (elosulfase alfa), developed by BioMarin Pharmaceutical Inc. is the enzyme replacement therapy for individuals with MPS IVA (Morquio A syndrome.)  Vimizim improved endurance in clinical trials and is administered weekly via intravenous infusion.

BioMarin will offer support to patients through its BioMarin Patient & Physician Support (BPPS) team. Through BPPS, patients receive live, personalized support by a specialized case manager who will research insurance coverage and alternative benefit options. BPPS will help patients obtain coverage and minimize out-of-pocket expenses and find alternative financial assistance for treatment. To reach a BPPS case manager, please call, toll-free, 1-866-906-6100 or e-mail bpps@bmrn.com. For more information about VIMIZIM, please visit www.VIMIZIM.com.

MPS VI

Naglazyme is the enzyme replacement therapy for individuals with a confirmed diagnosis of MPS VI and has been approved for use in the US and in many European countries. Developed and produced by BioMarin Pharmaceutical, Inc, Naglazyme has been shown to improve walking and stair-climbing capacity.

For more information, please contact BioMarin Patient and Physician Support (BPPS) at bpps@bmrn.com or 866-906-6100.

 

Please Participate in Short Market Research Survey Regarding ERT Infusions.

The National MPS Society has recently partnered with Engage Health, a market research company who is interested in learning more about the perceptions of patients and their caregivers regarding the administration of enzyme replacement therapy (ERT) infusions. The objective of this research is to better understand the logistics involved in getting infusions and patients’ thoughts about those logistics.

If you are a patient or the parent of a patient who receives ERT and are interested in participating in this short survey please click here. The survey will take less than 10 minutes to complete, and no preparation is required. As compensation for your time, a donation will be made to the National MPS Society or other patient association of your choice.

Thank you for your consideration.