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Rare Diseases Blog

By Mary Kugler, R.N., About.com Guide to Rare Diseases since 2000

TKT reports results of I2S trial for Hunter syndrome

Thursday June 30, 2005
On June 20, 2005, Transkaryotic Therapies, Inc. (TKT) announced the results of its Phase III clinical trial evaluating its drug iduronate-2-sulfatase (I2S) in the treatment of Hunter syndrome, also known as mucopolysaccharidosis II. I2S replaces an enzyme deficient in Hunter syndrome. In the trial, patients who received I2S on a weekly basis showed statistically significant improvement in a lung function test and in a six-minute walk test compared to patients receiving a placebo. TKT expects to file for regulatory approval of I2S in both the United States and Europe in the fourth quarter of 2005.

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