Very logical conclusion. I tend to agree that the 7 week trial may go for dosing - if there is any efficacy then good - else it will be used for safety establishment in young girls. The actual trial number announced initially was much higher
A collection of info from previous Rett trial announcements...and more This recent publication gives an idea why a drug targeting both S1R and M4 Muscarinic Receptor might be useful in Rett. We seem to be in for interesting times ahead.
Good analysis. Is the 15 patient study also described as double blind placebo controlled, or is that only describing the follow on study? Can you comment on that? A 15 patient placebo controlled study is important in that, if an efficacy signal is detected, though small N, it will have some legitimacy and withstand the scrutiny the 32 patient AD trial has suffered from. Your thoughts?