They have said some things at IR conferences but haven't given us hard data like for GALNS. They will have an Investor/Analyst Event in December which is supposed to focus on PEG-PAL. Basically we know there are at least some moderate injection site reactions and also patients with substantial efficacy. A daily injection with non-mild side-effects in this disease seems a harder sell unless they can completely liberalize diets IMO. My guess is they'll end-up with at least some subset that can liberalize diet and can justify a very high price.
I am actually quite excited at the prospects for 701 (Pompe) I've posted a couple of anecdotal patient reports on the BMRN board, the preclinical data always seemed to offer a much improved uptake and in the last IR conference JJ said it could be on the same timeline as PEG-PAL which I take to mean they could start a pivotal trial (late) next year (or maybe I am reading it wrong and PEG-PAL is even slower :-)). There is an interesting webinar from about a year ago but had some informative information like more specifics on the trial design, explained why going in Adults (seems obvious now but never heard at IR conference) and maybe another tid-bit or two http://www.amda-pompe.org/index.php/main/webinar/bmn-701_potentially_more_effective_treatment_option_for_pompe
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