Mucopolysaccharidosis Type VI
Is there a treatment(s) for Mucopolysaccharidosis Type VI?
The United States Food and Drug Administration (FDA) approved the use of a medication called galsulfase (also known as Naglazyme®) for the treatment of Mucopolysaccharidosis Type VI (MPS VI) in June of 2005. Galsulfase is an enzyme replacement therapy using a man-made version of arylsulfatase B, which is the enzyme that is not working properly in MPS VI. While galsulfase is not a cure for MPS VI, it helps reduce the amount of glycosaminoglycans building up in the body, which may help slow the disease process and improve symptoms.
- Maroteaux-Lamy Syndrome Resource Center - http://www.maroteaux-lamy.com/en-us/pages/patient/TreatmentERT.aspx
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What other things can be done to help the symptoms of Mucopolysaccharidosis Type VI?
Before treatment with galsulfase was available for people with mucopolysaccharidosis type VI (MPS VI), most treatment depended on a person's specific symptoms. A bone marrow or stem cell transplant can be used to treat some of the symptoms of MPS VI. The bone marrow produces blood cells and the theory behind the transplant is to remove the affected patient's bone marrow and replace it with the bone marrow from someone who doesn't have MPS VI. The white blood cells produced from this new bone marrow will be able to produce some arylsulfatase B. These transplants can improve the levels of arylsulfatase B in the body but do not improve the problems with the bones that people with MPS VI can have.
Are there other organ or symptom specific treatments for Mucopolysaccharidosis Type VI?
Mucopolysaccharidosis Type VI affects many systems in the body. Depending on the symptom to be treated, many different specialists can be involved, such as cardiologists (heart doctors), pulmonologists (breathing/lung doctors), medical geneticists (genetics doctors), ophthalmologists (eye doctors), audiologists (hearing specialists), otolaryngologists (or ENTs; ear, nose and throat doctors), neurologists (neurologists), and surgeons for any other health problems that may require surgical treatment. For example, there are orthopedic surgeries that individuals with MPS VI can have to relieve some of the pain and other complications from their bone problems. There are also some surgeries to remove the tonsils and adenoids that can help with some of the breathing problems like sleep apnea (where breathing stops or is very shallow during sleep, often caused by a narrow airway).