The trial did not meet its primary endpoint, a joint rank analysis of function and survival, and no efficacy was seen in the individual components of function or survival.

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In MPS I, a buildup of certain compounds in connective tissue leads to frequent infections, joint deformities and impaired heart function.

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The initial group of 1, 000 will participate in a series of baseline studies including cardiac function, testing for joint abnormalities and psychosocial tests.

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"A joint session of Congress has a different function, " Dallek said.

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