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Steady_T

01/27/22 9:35 PM

#347021 RE: georgejjl #347018

Thank you George. I miss read the updates Primary end points. Thanks for the correction. I'll redo or replace my post.

Steady_T

01/27/22 9:44 PM

#347024 RE: georgejjl #347018

Making it pivotal is a big deal and changing the primary outcomes is also a big deal.

The primary end point is Drug exposure-dependent response of the Rett Syndrome Behaviour Questionnaire (RSBQ) Total score. That is a behavioral description change.

The second one is Incidence of Adverse Events.

The secondary endpoint contain what may be considered the usual P2 PK measures and two more behavioral related scales.

There is nothing that could be considered biomarkers.




sab63090

01/28/22 7:25 AM

#347057 RE: georgejjl #347018

Georgeejil

Appreciate your comments, too! Sometimes I skim over your posts and think that they are just "promos", but you definitely have a clear understanding of the science, etc.

:}