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MPS and ML are genetic lysosomal storage diseases in which the body is unable to produce the necessary enzymes needed to break
down and recycle material in the cells. Because of this, materials are accumulated in the cells, inhibiting them from carrying
out their proper work. This can cause damage throughout all parts of the body that can continue to worsen with age.
Effects on the Body
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| A child with Hunter's syndrome (MPS II) |
MPS and ML can have many effects on all parts of the body due to the fact that the enzyme essential for breaking down materials
is absent from almost all the cells in the body. Seizures, glaucoma, hearing loss, airway obstruction, heart failure, diarrhea,
dental problems, and bone formation and growth problems are just some of the issues that those who have MPS/ML have to deal
with. Also, the nature of the disease causes deformities to the bones and tissue, the effects of which can be seen in the
picture on the left.
Treatment
As of now, there are no cures for MPS/ML, and so the effort of treatment is to improve the quality of life for the patient.
In some cases, bone marrow transplantation has helped, and enzyme replacement therapies are currently being tested for their
effectiveness. Otherwise, surgery to correct the deformities, as well as physical therapy to help with the joint problems
are pretty much all that can be done for now.
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