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Idursulfase Is Effective Treatment for Hunter Syndrome

ase infusions every other week. At enrollment the distance each patient could walk within 6 minutes was recorded, along with their forced vital capacity, which is a measure of how much air they can breathe in and then blow back out.

After 53 weeks, these measures were taken again. The weekly dosing group was able to walk, on average, 37 meters farther within 6 minutes than the placebo group. In addition, the weekly dosing group experienced an increase in forced vital capacity compared to the placebo group, and the weekly dosing group experienced more improvement on most measures than the every other week dosing group.

Liver and spleen size decreased by more than 20 percent after 18 weeks of treatment in both groups that received Elaprase, whereas liver size remained unchanged and spleen size increased in the placebo group. The groups that received Elaprase also saw a decrease in the amount of glycosaminoglycans (GAG) in their urine while the placebo group saw no change; greater levels of GAG indicate more severe disease. Finally, the groups that received Elaprase experienced an improvement in elbow mobility while there was no change in the placebo group.

Muenzer said anecdotal reports from the parents painted a more vivid picture of the improvement their children experienced.

“Before they began the infusions, these children typically could not walk through an airport or a mall without using a wheelchair. They would just get too tuckered out,” he said. “Afterwards, in a lot of these families, the kids ran through the airport and now the parents could barely keep up.”

(Source: Newswise)
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