Idursulfase (trade name Elaprase) is a medication used to treat Hunter syndrome, a rare metabolic disorder caused by the body's inability to produce a particular enzyme. It is a form of enzyme replacement therapy, in which the enzyme is delivered intravenously. Idursulfase can improve physical and cognitive function in those with Hunter syndrome, and may also help to reduce the rate of decline in lung function.
Idursulfase is a medication used to treat Hunter syndrome, also known as mucopolysaccharidosis type II (MPS II). It is a rare, genetic disease that affects males and prevents their bodies from breaking down certain complex sugars. These sugars build up in cells throughout the body, which leads to a variety of symptoms including coarsening facial features, an enlarged liver and spleen, stiff joints, and heart problems.
Idursulfase is a purified form of the enzyme iduronate-2-sulfatase, which is missing in people with Hunter syndrome. The medication is given by intravenous infusion (into a vein) once a week. It works by replacing the missing enzyme and helping the body to break down the complex sugars that build up in cells.
Idursulfase is typically well-tolerated, but some people may experience infusion-related reactions, such as fever, chills, headache, and fatigue. These reactions are usually mild and go away on their own within a few hours of the infusion.
Idursulfase is not a cure for Hunter syndrome, but it can help to improve symptoms and slow the progression of the disease.