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Another from SpeedyAgain, kindness of Speedbll…(who ought to post here) ================================================================== "Key takeaways from RBC Conference Call... DI is very pleased that PFE seemed to show some signs of efficacy with their lousy cassette but you gotta be NUTS if you’re gonna subject a kid to a capsid dose over three times SRPT’s, with crummy expression and that almost killed half the kids that took it. And it’s just a little suspect when PFE took biopsies and did Western Blot then argued that Western Blot is a poor metric. Succinctly, AAV9 therapies for DMD ARE LOUSY DI doesn’t know if the high dose LGMD2E patients will have higher expression than the lower dose but, by the way, this high dose is the same dose as the DMD study and there could be a lot of read thru supporting the DMD dosing but, you know, we don’t really know if this LGMD2E dose will be any better, but, you know of course, that we do know that higher is better, and we did dose three times higher than this preclinically, and, like we said, higher expression is better because this is a once and done therapy but we’ll just have to wait and see and we did already take all the biopsies but we kept our eyes closed and did it in a really dark room so we don’t have any idea if we got phenomenal expression like we did with the same 2e to the 14 dose that we made history with in the MicroD study... ((Speedy here:)) Yeah, ok. In other words, the LGMD2E high dose results are EXCELLENT" |
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