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I am still long. Actually the Pompe setback was a bigger disappointment for me. As I thought that to be their best market opportunity (Genzyme's ERT(s) leave a lot to be desired and no other near term competition). Not just the initial AE's from (what they claim was too high a dose) but going back into Phase 1. However, I am not quite as negative on the company/technology as genisi :-). I think in one of Shire's calls a while back they characterized the technology well as being (still being very early and several years away).
One big problem is I did not see them at being cash flow positive even with Amigal approval (though it would certainly buy them a lot of time). The long argument is a bet on another deal of some sort, e.g. Shire, Genzyme or BMRN decide their chaperone with ERT offers a superior product (not to mention better IP) so they cut some sort of deal to combine the two.
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