MPS Genetic Centers 

MPS I Aldurazyme 2014 Co-Pay Assistance Program

New in 2014:

•In addition to providing co-pay assistance for eligible out-of-pocket drug and infusion related co-pays, co-insurance and deductibles, Genzyme will also provide assistance for mixing of the drug as well as infusion supplies such as saline, IV tubing, syringes, etc.

•Please note that infusion-related (including supplies and mixing of the drug) co-pays, co-insurance and deductibles are NOT reimbursable for commercially insured patients who reside in MA, MI, MN or RI in accordance with State law.

•Patients who are currently enrolled in an insurance plan through the Health Insurance Exchanges are eligible to enroll in the Genzyme Co-Pay Assistance Program.

•Patients who are currently enrolled in a Federal Employee Plan as well as Postal Service Workers are eligible to enroll in the Genzyme Co-Pay Assistance Program.

•Patients who are currently enrolled in Genzyme’s Charitable Access Program (regardless of where they reside) are eligible to enroll in the Genzyme Co-Pay Assistance Program for assistance with their infusion-related costs.

•Patients who are currently enrolled in the program will not need to re-enroll as this will be done automatically on an annual basis and ID#s will remain the same.

•Patients will be given the option to opt out (un-enroll) from the program at any time if they so choose.

•New enrollment cards will only be mailed out to new enrollees in 2014; existing enrollees will keep the same enrollment card

MPS I Registry

Access to information is critical to providing the best care to patients with MPS I however information on the disease is limited because of its rarity. Now there is a resource which your physician or health care professional can use which is dedicated to improving the understanding of MPS I disease. Ask your physician to call 1.800.745.44471.800.745.4447 ext. 17021 for more information.

MPS I Website

A Web site has been developed by Genzyme to provide parents and patients with information and resources on MPS I. This site provides valuable information on the disease, diagnosis, on-going clinical trials, and other references and services available to patients. Visit

Aldurazyme Website

A website has been developed by Genzyme to provide parents and patients with information on Aldurazyme. The site includes a link to ask questions regarding MPS I or anything else related to treatment. Feel free to use this mechanism to reach a healthcare professional at Genzyme who will respond to your query in a timely manner.Visit

Genzyme Treatment Support Offers MPS I Families Insurance Counseling

Genzyme Treatment Support is a free and confidential service staffed by a team of nurses, social workers, and other health care professionals who act as patient advocates and provide confidential one-on-one insurance counseling to patients and their families. For more information, call 1.800.745.44471.800.745.4447, Monday through Friday.

MPS II Intrathecal Clinical Trial Update

The Phase II/III trial of idursulfase-IT in MPSII patients with cognitive impairment (HGT-HIT-094, AIM-IT study) has been on Complete Clinical Hold in the United States as the FDA requested additional information on the intrathecal drug delivery device (IDDD).  On January 9, 2014, AIM-IT has been moved from Complete Clinical Hold to Partial Clinical Hold Status.

MPS II Website

Shire HGT educational Web site focuses on MPS II (Hunter syndrome). The site,, is a resource center for the MPS II community to access information about the genetics, diagnosis, and management of MPS II, as well as information about the drug development process. In addition, the Website provides a comprehensive overview of MPS II, including resources for patients and healthcare professionals, information on clinical trials and a patient outcomes survey, as well as the ability to stay informed as new information about MPS II becomes available on the site. Shire HGT expects to update and expand the site on a regular basis.

Shire HGT Offers Support for MPS II

Shire Human Genetic Therapies introduced OnePath support center to help understand the complex issues pertaining to ERT, coverage, coding and reimbursement. When you call OnePath, youll be assigned a personal case manager who will help address any questions or concerns associated with ERT. OnePath case managers have access to many important resources; they also get to know you and can anticipate your needs. OnePath case managers can also address questions about insurance and other issues related to ERT. Toll-free number: 866.888.0660866.888.0660

BioMarin Patient & Physician Support Team Offers Support for MPS IVA

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, call, toll-free,866.906.6100 or e-mail

Learn more about the people who make up the Morquio A community, discover helpful online resources, and create a list of questions to bring to your next doctor’s appointment.

Enzyme Replacement Therapy for MPS IVA

Discover how VimizimTM, the enzyme replacement therapy for MPS IVA, works, read success stories of individuals currently taking Vimizin, find a geneticist with MPS IVA experience near you, read tips on how to talk to your doctor about Vimizin, and learn how to get access to this new treatment.

MPS IV Registry

Information about MPS IV can be found at Also available on this website is the Morquio registry where adults with MPS IV can register and families can register their child with MPS IV. Once registered, it is recommended that updates be made at least yearly. This natural history information is critical for development of treatments for MPS IV, providing evidence of drug effectiveness and supporting the approval of the drug.

MPS VI Website

BioMarins Web site,, is designed especially for individuals with MPS VI (Maroteaux-Lamy syndrome), their families, and for healthcare professionals who care for patients with MPS VI. This site provides education and information about MPS VI which may be helpful to share with family members, educators and healthcare providers.

MPS VI Community Website

Log into the first Web site devoted entirely to the MPS VI community and:
Meet other people with MPS VI
Tell your story
Chat in real time
Search postings by topic
Register for free to connect with your MPS VI community.

BioMarin Offers Help with Insurance Questions for MPS VI

BioMarin has developed a free and confidential service designed to assist with healthcare insurance questions. The BioMarin Patient and Physician Support program (BPPS) can help families evaluate their current healthcare insurance coverage, provide information on potential healthcare insurance options that may be available in your state, and educate insurance companies about MPS VI. To contact a BPPS patient advocate, call their toll free number 1.866.906.61001.866.906.6100.

Health Care Coverage Options

The National Association of Health Underwriters has created a database that U.S. residents and people who change jobs or have pre-existing health problems. The Health Care Coverage Options Database provides a state-by-state list of public health insurance programs for low-income residents, such as Medicaid and the State Childrens Health Insurance Program. It also lists 32 states that offer high-risk health insurance pools for those with pre-existing health problems.

Transitions: Managing Your Own Healthcare – What Every Teen with an LSD Needs to Know”

Click here for the book: “Transitions:  Managing Your Own Healthcare – What Every Teen with an LSD Needs to Know.”

This book is intended to help families successfully manage the transition from childhood to adulthood in terms of learning how to take over your own healthcare management and living with a lysosomal storage disease.  Please share it with any MPS teens, preteens, and parents!