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Re: mcbio post# 212089

Monday, 06/26/2017 8:53:33 AM

Monday, June 26, 2017 8:53:33 AM

Post# of 252311

What about their PPMO program? They have talked that up quite a bit. I believe it is set to enter clinic by end of this year. Do you think that can overcome the issues that you predict will plague etep and the other related more advanced exon skipping programs?



No idea. I don't routinely track SRPT for the obvious reason that, as the FDA documents revealed, they have a horrendous history of being very misleading. Also note that so far we have no results from the next gen oligos, which allow much lower dosing since they are better at staying at the target organs. (It'll be 3 or 4 years until we have big, long duration, RCT data for one of these drugs)

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