Current Research into MPS I

There is at present no cure for MPS I. However, many centres around the world are furthering research into this rare disorder.

In some parts of the world, there is a history of using Bone Marrow Transplantation (BMT) as a way of getting the missing enzyme into the body of MPS patients. This has been shown to be beneficial in younger children with MPS I before the onset of severity of symptoms. However, BMT does not seem to be performed as commonly here in Australia.

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 I. Currently there is a clinical trial for MPS I being conducted in five centres around the world. The results of this will hopefully be available soon.

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.

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