Enzyme replacement therapy

Enzyme replacement therapy (ERT) uses an intravenous solution (IV) to replace a deficient or missing enzyme in the body. ERT does not cure the disease but slows its progress by increasing the amount of missing enzyme in the body.

Enzyme replacement therapy is currently available for four MPS disorders MPS I, MPS II, MPS IVA and MPS VI.

MPS Enzyme Replacement TherapyMPS I

Aldurazyme™ (laronidase), administered once weekly, is approved in more than 60 countries worldwide, including the United States and across Europe 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 SanofiGenzyme under a joint venture agreement that assigns commercial manufacturing responsibilities to BioMarin, and worldwide sales and marketing responsibilities to SanofiGenzyme.

Visit www.aldurazyme.com or contact SanofiGenzyme at 800-745-4447 for more information.

MPS II

Elaprase™ (idursulfase) is a long-term enzyme replacement therapy for patients with a confirmed diagnosis of MPS II and has been approved for use in the U.S., Canada and many countries in Europe. Elaprase was developed and is produced by Shire Pharmaceuticals and is given as a weekly infusion.

For more information, visit www.shire.com or contact OnePathSM at 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 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 866-906-6100.

For more information about VIMIZIM, visit www.VIMIZIM.com.

MPS VI

Naglazyme™ (galsulfase) is the enzyme replacement therapy for individuals with a confirmed diagnosis of MPS VI and has been approved for use in the U.S. 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, contact BioMarin Patient and Physician Support (BPPS) at bpps@bmrn.com or 866-906-6100.

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The National MPS Society exists to cure, support and advocate for MPS and ML.

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