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Idursulfase Is Effective Treatment for Hunter Syndrome

g of facial features and skeletal deformities, developmental delays and neurologic disease, and, ultimately, an early death. Muenzer estimates there are only about 500 people with Hunter syndrome in the United States.

“We’re pleased that ELAPRASE, the first approved treatment for Hunter syndrome, is now on the market and available to patients,” said David Pendergast, president of Shire Human Genetic Therapies. “Dr. Muenzer played a key role in the clinical trials of this drug, leading to successful FDA approval.”

Sixteen-year-old Cody Paxton, who was the first patient enrolled in the Phase II/III trial at UNC Hospitals, says the weekly, 3-hour infusions of Elaprase have helped him tremendously since he started receiving them in September 2004.

“My breathing is better, and I’m more energetic,” he said. Plus, Cody said, he has much more flexibility in his joints. Now he can place a hand behind his head, which he could not do before, he said.

Cody and his mother, Elena Paxton, moved to North Carolina from Mastic Beach, N.Y. so Cody could take part in the clinical trial. Cody’s older brother, Christopher Dutcher, who is now 22 years old, was the first patient enrolled in the earlier Phase I/II trial at UNC Hospitals.

“His liver shrank within 3 months after Christopher started receiving the infusions,” Elena Paxton said. “His air intake got better and he had more energy. Plus, Christopher has remained relatively healthy, instead of continuing to deteriorate,” she said.

The treatment has worked so well for him that Christopher, who still lives in New York, was able to earn a college degree and will begin graduate school in the fall at Hofstra University, his mother said.

Muenzer’s article reports that 96 Hunter syndrome patients between 5 and 31 years old were enrolled in the clinical trial. Some received placebo infusions, some received weekly infusions of Elaprase and some received Elapr
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