MPS VI Registry

The Women's and Children's Hospital in Adelaide and BioMarin Pharmaceutical (USA) are pleased to announce the establishment of a web site Registry dedicated to patients affected by MPS VI (Maroteaux-Lamy syndrome). 

What is the purpose of the web site Registry? 

MPS VI is a rare disorder.  As a result it has been difficult to gather important information about the disorder in an organised way.  The Registry's primary purpose, therefore, is the creation of a world-wide database of people affected by MPS VI that provides information to doctors, researchers and patients about its prevalence, its natural progression, the range and severity of clinical symptoms, and up-to-date information about research results, clinical trials and new treatments.   The web is an ideal vehicle for such a project. 

What is the relationship between WCH and BioMarin for the purposes of the Registry? 

The Adelaide Women's and Children's Hospital (WCH) and BioMarin Pharmaceutical have entered into a special arrangement to establish this Registry. 

The project supervisor at the WCH is Professor John Hopwood.  Professor Hopwood's group is a leader in researching diagnostic and treatment methods for MPS VI.  The WCH is involved in the Registry because of its long-term interest in MPS VI, and it believes the Registry is an important resource that will contribute a great deal to our knowledge about MPS VI to the ultimate benefit of patients.  WCH's participation in the Registry has been approved by the WCH Ethics Committee.  

The web site and Registry database are owned by BioMarin Pharmaceutical, Inc.  BioMarin is based in California, USA, and is conducting clinical studies for the treatment of MPS VI.  The information contained in the database is owned by BioMarin.  BioMarin is contractually obligated to the WCH not to access individually identifiable patient data, nor make it available to others who may have an interest in obtaining the data.  BioMarin is bound by this obligation on an on-going basis. 

The WCH will be involved in the Registry data collection for a period of 12-months.  After this initial establishment phase, BioMarin intends to transfer the database and the task of on-going data collection to a non-profit organisation dedicated to alleviating suffering of MPS-VI patients.  This organisation will be under the same obligations of confidentiality as the WCH.  BioMarin will notify all patients registered in the database prior to the transfer of the data.

What information will be collected? 

Individuals who register will be asked to answer a series of general medical questions relevant to MPS VI.  They will also be asked to provide their names, addresses and contact numbers.  Contact details for their physician will also be required.  The results of laboratory or other tests will not be included in the Registry. 

Who will have access to the information? 

The only people who are permitted access to information that identifies patients will be the Registry Coordinator, Professor Hopwood, and a member of Professor Hopwood's group, all of whom are employed by the WCH and are directly involved with this project.  These individuals are under strict obligations of non-disclosure.  Each individual who registers will assign themselves a password to access their own data only. 

BioMarin will not have any input into, or be involved in, data collection.  BioMarin will have access to the information to enable on-going maintenance of the web site. 

Will the information be published? 

It is possible that the information collected in the Registry may be published in scientific journals.  However, individuals will not be identified in any publications that may arise. 

How secure is the web site? 

Strict security measures have been incorporated into the design of the web site by an independent third party, and both the WCH and BioMarin are under strict obligations of confidentiality and non-disclosure, neither is able to guarantee that the site and its contents will not be breached by unauthorised users (i.e. 'hackers').  For added security, three levels of access have been incorporated into the site: public, research and confidential. 

• Public access is available to anyone who enters the site.  From this level of access, medical information can be viewed, but only in anonymous form. 
• Research access is the same as public access, but will enable researchers with Ethics Committee-approved projects to access the contact details of doctors (but not patients).  This will allow them to let doctors know about the latest research findings, which may be of benefit to their patients. 
• Confidential access is available only to the Registry Coordinator and two other staff at the WCH.  This level of access contains identifiable patient information. 

Are there any costs involved? 

Participation in the Registry is free of charge. 

Can patients withdraw their information from the Registry? 

Patients have the right to withdraw their participation at any time by completing the appropriate document on the web site. 

Who can register, and how? 

Anyone affected by MPS VI can register by accessing the web site https://www.mpsvi.org/Registry/index.cfm

 Patients will be asked to identify the physician who manages their symptoms associated with MPS VI.  The physician will then be required to verify the information provided by the patient before registration will be deemed complete.  Patients under the age of 18 years will require the approval of their parent/guardian before being able to register.  Once the process of verification is complete, the Registry Coordinator will 'de-identify' individual patients by the use of a special code number to maintain confidentiality and privacy.  This means that anyone entering the web site will be able to see the medical information, but not be able to identify an individual patient. 

The web site outlines the Terms and Conditions of Use and provides a Privacy Statement which details what information is being collected, circumstances under which it might be released and the measures in place to ensure its security. 

If internet access is not available, the required documents can be obtained in paper form by contacting the Registry Coordinator on the appropriate toll-free number listed below. 

Further questions? 

Special toll-free numbers have been established for people to obtain more information about the Registry from the Registry Coordinator. Please note that English is the preferred language for the use of this service. 

Voicemail has been arranged for calls that may be received outside of normal business hours.  In this circumstance, please leave your name, contact telephone number and the country you are calling from, and the Registry Coordinator will return your call on resumption of business the next day. 

Contact Details: 

MPS-VI Registry Coordinator

Department of Chemical Pathology Women's and Children's Hospital 

72 King William Road 

NORTH ADELAIDE, SOUTH AUSTRALIA 5006 

AUSTRALIA. 

Fax: (08) 8161 7100 

International prefix: (61 8) 

Email: oatess@senet.com.au 

International toll-free numbers: 

WEB-BASED PATIENT REGISTRY FOR MUCOPOLYSACCHARIDOSIS TYPE VI (Maroteaux-Lamy syndrome) ] 

Women's and Children's Hospital, Adelaide, 

BioMarin Pharmaceutical USA

Back to top