Current Research Into MPS II

At present there is treatment for symptoms as they arise, but no cure for MPS II. Various experimental methods have been used to try and replace the missing enzyme, but none has so far been of any significant long-term benefit.

Bone Marrow Transplantation (BMT) has been shown to be largely ineffective for the treatment of Hunter syndrome.

The most promising therapy to date may be Enzyme Replacement Therapy (ERT). ERT refers to the infusion of manufactured enzyme into a patient's body to enable the stalled recycling process to begin and work normally. Experience with Gaucher Disease indicates the benefit that ERT could have for other lysosomal disorders, including MPS II. A clinical trial for MPS II is expected to begin soon and we look forward to the results.

The Lysosomal Diseases Research group has as its mission the early detection and treatment of lysosomal storage disorders (LSDs). It is hoped that in the future LSDs will be diagnosed soon after birth and so enable the effective use of therapies before the onset of clinical symptoms.

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

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