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Re: Doc328 post# 349189

Friday, 02/04/2022 1:04:52 PM

Friday, February 04, 2022 1:04:52 PM

Post# of 462736
Good information, Doc. I can’t tell if you’re long, short or neutral / no vested interest in AVXL as an investment but I always appreciate your insight.

My response to your post is general agreement, but do you think if there is an unmet need for a relatively rare disease without any approved treatment the FDA may be more willing to permit a smaller trial given the drug’s positive safety profile?

Could it approve Rett now on a provisional basis with the pediatric study serving more as a P4-style trial to confirm the data? It seems that if the Rett community applies enough pressure the FDA may cave. We saw with Biogen’s AD drug that the FDA is willing to act outside of the norm when there appears to be a significant unmet need.
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