Penny Double...thanks for this insight. We do understand that the last n* decades in CNS disease trials & research have not resulted in very many we know as AAHHA moments.(try none) Smart people, working hard getting about nowhere, except they know what does not work. Experimenting for what does not work is a painful way to learn as one quickly runs out of $$$ and time. IMO, time turns out to be the biggest issue, we all only have so much of it. This compression should mean we do multi tasking/trials but guess what, the same idiots who keep looking for placebo numbers have likely never run a trial or had a true appreciation for time to achieve results. Knowledge and the learning process is what really matters, however that is done.
So then, here we are. We a have a strong history of producing CNS disease trial results that prove what does not work. IMO, the AVXL team gets it. They have managed for time by doing multiple trials simultaneously on different CNS diseases. They have selected exactly what they want to prove. They have isolated and identified the critical variables using biomarker and other methods. They know exactly what they are doing and the trials form/structure is almost not relevant. Getting results is what matters. If these kids get up and walk out of RETT trials beds then we win. The rest of it is details which we will easily manage. Paying attention is tough when you are learning as you go that an alternate path will get best results, and lying is not an option. Thanks again for letting me babble.
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