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Re: LakeshoreLeo1953 post# 122622

Saturday, 09/30/2017 10:44:30 AM

Saturday, September 30, 2017 10:44:30 AM

Post# of 458440
My time with my boys is considered the most sacred time for me - and hence, it always suffers since I always want to be around my two sons.

I'm not sure of your question, leo, so here is my best attempt.

What I am looking for only in Retts is - statistically significant reduction in seizures compared to baseline seizure frequency (relative to placebo control). I am also looking for improvement in coordination.

If Rett trial shows a significant reduction in seizure frequency - this will serve, in my opinion, as a gateway to a much broader epilepsy indication and pave the way for breakthrough designation or accelerated approval for the other orphan drug designations of fragile x syndrome and possibly infantile spasms. With these approvals - the race to broader indication of "partial onset seizures" (or partial epilepsy/focal epilepsy/localization-related epilepsy) would then start - with quicker time to market for epilepsy than what we think of it now. I foresee an epilepsy indication leapfrogging over an AD indication for FDA approval IF the REtt trial is successful.

The Rett trial is much more important that people currently think, in my opinion. IT will be the "make or break' trial for this company and hence, why the design and planning of it is taking so long. With success - the possibilities for other epilepsy-related indications become abundant. With failure - all the other possible epilepsy indications will take longer, if at all, to come to fruition and optimism for quick AD and PD trials will be stymied.

Hope this sheds some light onto how I think of the current position long investors like myself are in.

Hope all is well Leo!
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