Morquio FAQ's
What causes the disease?
Mucopolysaccharides are chains of sugar molecules used to build connective tissues in the body.
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“muco” refers to the thick jelly-like consistency of the molecules.
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“poly” means many.
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“saccharide” is a general term for a sugar molecule.
The body constantly replaces used materials and breaks them down for disposal. MPS IV patients are missing an enzyme essential to breaking down the mucopolysaccharide keratan sulfate. These materials remain stored in the body’s cells, causing progressive damage. Babies may show little sign of the disease, but as cells sustain damage, symptoms start to appear.
How is this disease inherited?
MPS IV is caused by a recessive gene. There is a one in four chance with every pregnancy that the child will inherit the defective gene from each carrier parent and will be affected with the disease. There is a two in three chance that unaffected brothers and sisters of children with MPS IV will be carriers.
Is there a cure for MPS IV?
There is no cure for MPS IV. In 2014, Vimizin was approved by the FDA as an enzyme replacement therapy (ERT) for MPS IVA. There are clinical trials moving towards gene therapy and improved ERT.
Family Planning
Preimplantation genetic diagnosis (PGD) is a procedure used during fertility treatments to help identify genetic defects within embryos. This serves to prevent certain genetic diseases, such as MPS, from being passed on to the child.
