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Re: Investor2014 post# 451624

Sunday, 02/11/2024 2:13:18 AM

Sunday, February 11, 2024 2:13:18 AM

Post# of 461850
To use your argument, the heightened expectations of the placebo group should be the same for the treatment group. After all, it is unlikely that the subjects knew which arm they were in. If you claim that the kids and care givers in the treatment group were able to figure out that they were in the treatment arm then it would seem that they had lowered expectations compared to the folks that had not figured out which group they were in i.e. the placebo group.

The reason given by the company for the strong placebo response ascribed it to different levels of disease severity between the two arms. The random selection process when selecting small numbers from a larger population can sometimes result is a less representative group being selected. A larger selected group tends to generate a more representative group of the larger population.

Validated biomarkers for Rett would be very helpful in this regard, as you say. Unfortunately the genetic changes found between the treatment group and the placebo group are not yet validated biomarkers. At this point they sure are suggestive.

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