Anavex Life Sciences Corp (AVXL)

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Percy is an investigator for both the Anavex and trofinitide studies. He is likely well connected in the academic Rett community as a center of excellence clinic director for many years. My takeaway from the article is that he is optimistic current drugs in trials may succeed but he feels the RSBQ is not a great scale. It only captures behavior issues but not the very important physical symptoms and their progression. He has seen both drugs in action and i wonder if he notes mild improvements but nothing that drastically changes the life of the child or family.

I think Anavex has a good chance be approved eventually (assuming Excellence outcomes are excellent). How much of a difference will these drugs actually make. Are any of the girls talking? Are they having less scoliosis surgery or walking better? Are the girls with feeding tubes now eating? And Importantly for economic modeling, are fewer healthcare and support personnel dollars being spent. Should society pay 200,000 a year for a girl to wave their hands less and sleep mildly better but still require 24/7 supervison? I wonder how ICER would rate the benefit.