Hurler syndrome

Preliminary results in mice appear encouraging for rare conditions like hemophilia B, Hurler syndrome, Fabry disease and Wolman disease.

The proposal would require the Department of Health and Environmental Control to test for Hurler syndrome, Krabbe and Pompe disease in addition to the 53 conditions the agency already screens.

Sangamo is also testing the liver-factory gene treatment in trials now getting underway for the blood-clotting disorder hemophilia B and another metabolic disease, Hurler syndrome.

Further safety trials using the same technology to treat haemophilia B and Hurler syndrome are also planned.

Jacob, whose family lives near Lake Goodwin north of Marysville, was a baby when he was diagnosed at Seattle Children’s Hospital with Hurler syndrome.