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Talon38

03/12/18 5:14 PM

#144516 RE: Rubyred77 #144485

Ruby, good article and shows how far we have advanced in our drug development thinking. It's a great credit to Scott Gottlieb and Janet Woodcock for much of this new trial architecture. The Rest Syndrome Foundation Natural History Study has been on going since late 2014. There is obviously much data around which Anavex, the RSF and the FDA/CDER have developed the current P2 trial.

Whether CDER is incorporating this into a "basket trial" for the other Orphan Diseases is yet to be seen. I personally don't think that a basket trial approach should delay the RS trial as the other diseases can be phased in or run sequentially and benefit from the data coming out of the RS trial.

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https://www.rettsyndrome.org/research/clinical-trials/natural-history-study