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jq1234

07/23/12 7:56 PM

#145915 RE: turtlepower #145909

There are a small percentage of patients who don't tolerate ERT that well, there are also patients whose symptoms are mild enough decided not to be on ERT anymore for variety of reasons. You don't have data on patients who were on ERT successfully then switch to Amigal immediately. Those switches usually are monitored very carefully, and need to be in separate trials, because they could distort trial results if mixed with treatment naive patients (discontinued ERT for 6 months or longer are considered treatment naive, so really doesn't matter how the breakdown was). They have an EMA trial where patients were on ERT successfully for at least one year. Data in 2014.