Pompe Program Update

Category: Archive
September 13, 2005

This is an exciting time for Pompe patients and caregivers. The BLA (Biologics License Application) was filed with the FDA on July 28th, and it is expected that there will be a formal response to this filing in early 2006, as well the European filing to the EMEA, in the first half of 2006.

The Late-Onset Treatment Study is now enrolling patients at sites in the US and Europe, and by early 2006, it is expected that the number of individuals with Pompe disease receiving the investigational rhGAA enzyme will be approaching 200.

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