Although most patients with MPS IVA generally appear healthy at birth, patients often show skeletal deformities within a few years of age including scoliosis, knock-knees, chest wall deformity, arthritis and short stature. Over time, children may develop additional symptoms including sleep apnea, hearing loss, visual impairment, enlargement of the liver and spleen, dental abnormalities, impairment of the heart valves and compression of the spinal cord. MPS IVA patients often become severely handicapped and become wheelchair bound when they are teenagers. Patients with a severe form die of respiratory problems, cervical spinal cord complications, or heart valve disease in their 20s or 30s if untreated.
In the US and in most other developed countries, children like Jonathan, Desmond and Charity could be receiving an enzyme replacement therapy drug that helps restore cell function. The therapy improves health and quality of life significantly in some patients. It is, however, is a very expensive drug, and out of the question for patients in low-middle income countries.
PROS has been able to provide power wheelchairs for these 3 children so that they can attend school without assistance and is working to raise money to pay for the drug treatment for these children and for other patients in Africa that can’t afford therapies. Please consider a tax-deductible donation.
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