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Re: turtlepower post# 134936

Thursday, 01/12/2012 7:41:13 PM

Thursday, January 12, 2012 7:41:13 PM

Post# of 252254
Fold: no special thoughts on the trial. Maybe interest due to ert shortages.

I actually like the idea of chaperone+ERT just more for Pompe where need to get better uptake in some cells. I haven't looked at interim data in much detail to speculate on how good it is. Saw very small patient numbers. Genisi is better person to analyze that too.

For Fabry I thought best opportunity for late onset less symptomatic patients who may not want hassle of ERT but this will take a lot of effort to reach/penetrate.


Sorry for writing in phrases on iPad.

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