Current Research into MPS VI

There is no cure at present for MPS VI. Bone marrow transplantation (BMT) may be helpful in some patients and there are cases where it has improved life-threatening airway obstruction. At this stage BMT is an ineffective treatment for the bone disease present in MPS VI.

Current research into MPS VI is occurring in Adelaide. Research is focused on ways of getting the missing enzyme into bone using a naturally occurring animal model in Siamese cats. Positive results have been noted including Improvement in bones and growth when BMT occurs early.

Research Report from Biomarin on MPS VI: 

Currently MPS VI (Maroteaux-Lamy Syndrome) patients are being enrolled in a survey study designed to collect much needed information about the range and severity of clinical symptoms in MPS VI. The study involves 2-3 days of clinical evaluations at one of four centres located in the US, Germany, UK, and Australia. For additional information, please contact Dr. Paul Harmatz, Children's Hospital Oakland (pharmatz@chori.org).  

Following the positive results of the Phase I enzyme replacement therapy (ERT) trial for MPS VI, a Phase II trial of ERT will be starting soon. For additional information about the initial trial or the Phase II trial please contact Dr. Paul Harmatz, Children's Hospital Oakland (pharmatz@chori.org) or visit the BioMarin web site at www.biomarinpharm.com.

Further information

More information on MPS VI is available from our National Office, including an MPS VI syndrome booklet which can be purchased with the enclosed order form. 

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