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Investor2014

01/05/24 9:17 AM

#446146 RE: boi568 #446135

I still think you forget to consider that the placebo vs drug arms are controlled for ALL characteristics of the trial including the mechanics of how it is conducted.
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Kentucky123

01/05/24 9:27 AM

#446153 RE: boi568 #446135

Why have CGI as an endpoint if it is useless? Poor trial design. They blew it. Move on to AD.
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bas2020

01/05/24 10:37 AM

#446185 RE: boi568 #446135

To your point, it's more about subjectivity and perhaps bias of the reviewer than it is an actual placebo effect in the patient... who, in the case of Rett, are likely unaware they're receiving a drug/placebo. Also, the reviewers may be hearing biased comments from the caregiver/parent during the evaluation.

Biomarker data will bring objectivity into the evaluation (e.g. gaba & glutamate), as well as QOL improvements, including sleep and reduction in seizure frequency.