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new treatment for Hunter Syndrome (MPS II)

[Hunter Syndrome is in the same group of diseases as San Filippo Syndrome. San Filippo Syndrome is MPS III.]

On Monday, July 24, 2006 Shire Human Genetic Therapeutics announced that Elaprase™, the enzyme replacement therapy for MPS II (Hunter Syndrome), was approved by the FDA.

Hunter syndrome is a rare, life-threatening genetic condition that results from the absence or insufficient levels of the lysosomal enzyme iduronate-2-sulfatase. Without this enzyme, cellular waste products accumulate in tissues and organs, which then begin to malfunction.

ELAPRASE is the first and only treatment approved for people suffering from Hunter syndrome. The product, which is given as weekly infusions, replaces the missing enzyme that Hunter syndrome patients fail to produce in sufficient quantities.


“Regulatory approval of ELAPRASE will enable physicians to move needy patients beyond palliative care and make Hunter syndrome a treatable disease,” said Joseph Muenzer, MD., Ph.D, of the University of North Carolina at Chapel Hill. “Until today, there were no options for addressing the underlying cause of this devastating disease.”

Shire expects to launch ELAPRASE in the United States within the next 30 days.

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