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OFP

Re: Investor2014 post# 140900

Tuesday, 02/13/2018 9:32:11 AM

Tuesday, February 13, 2018 9:32:11 AM

Post# of 517137

Why base the dosing of Rett girls on mouse data when we have data from multiple dosing schemes in humans?

I suspect they did try to base it on human data with using the PK data to target blood levels that were associated with better response in the P2a AD trial. The FDA rejected on the basis that that dosing level was not tolerated by the majority of subjects in that trial. Just to anticipate...that is all speculation.

They could have used mouse data as well but the equivalent doses would have been even higher and therefore likely even more out of reach.
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