Metabolic diseases: New drug slows down Hunter's disease

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Metabolic diseases: New drug slows down Hunter's disease
Metabolic diseases: New drug slows down Hunter's disease

New drug slows down Hunter's disease

Elaprase, a new drug for Hunter Syndrome, has been successfully tested at the UNC School of Medicine in Chapel Hill. With it, a highly effective drug against this deadly hereditary disease could be available for the first time.

In their study, Joseph Muenzer and colleagues tested the effect of the drug on 96 patients. After a year, the typical Hunter indicator values in the urine as well as the elbow mobility and the ability to walk of the patients improved in those treated. They were able to walk an average of 37 meters further in six minutes than patients treated with a placebo. Some children, who otherwise would not have been able to do without a wheelchair, were even able to get out of it and take a few steps thanks to the therapy. In addition, disease-related spleen and liver enlargement decreased by more than twenty percent in just 18 weeks.

Elaprase contains the enzyme idursulfase, which is missing in Hunter's disease patients and which the body needs to recycle complex sugar compounds. Instead of being subsequently used, the compounds are deposited in the diseased person in various tissues and organs of the body.

The approval process for Elaprase is still ongoing in Europe. According to Gudrun Schulze-Frenking from the University Clinic in Mainz, approval can be expected by the end of 2006.

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