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Hematology Week

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Gene Therapy



New gene therapy research from Northwestern University, Department of Pediatrics discussed



April 16th, 2007

Investigators publish new data in the report "The effect of enzyme replacement therapy on bone crisis and bone pain in patients with type 1 Gaucher disease." According to a study from the United States, "The effect of enzyme replacement therapy (ERT) on bone crisis and bone pain was investigated in patients with Gaucher disease (GD) type 1 followed over 4 years. Data from the International Collaborative Gaucher Group Gaucher Registry were used."

"Only patients with bone crisis and/or bone pain data for 1 year prior to ERT, and for each of 3 years after the start of ERT, were included. Bone crises were reported in 17% of patients during the year before starting ERT. The...


Source: Hematology Week (2007-04-16)

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