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scratchy

03/01/21 12:09 PM

#301175 RE: AceT #301174

Thanks AceT

oldandintheway

03/01/21 12:13 PM

#301177 RE: AceT #301174

Well done AcT

hnbadger1

03/01/21 12:14 PM

#301179 RE: AceT #301174

He also briefly spoke about the recent undisclosed new trial, and how they were working with the parents and the advocacy’ group.

nidan7500

03/01/21 12:30 PM

#301183 RE: AceT #301174

AceT:

One example CM mentioned was that during the Rett planning the discussions provided insight on how tough the mobility can be for that community so they designed the trial to allow for more in home visits, blood draws for example, that would limit the travel needs. This proved to be very beneficial during Covid.

Nadia - Advocacy groups and community ecosystem groups can provide different insights and recommended to interact with both during the planning phases.

CM - We have found that making the patient the focus instead of the compound the focus allows us to design better trials for all.



Great catch. I am very hopeful that this kind of science/human interface mix was also allowed in the practice of PDD Actigraphy data gathering methods and practices.

I remain grateful that Dr.M. and AVXL team have remained focused and insightful of , "just why are we doing this", as they plan and execute clinical trials.

Paying close attention to trial execution context (w/trial subjects) can help to avoid misunderstandings and errors in interpretation.

Investor2014

03/01/21 12:43 PM

#301186 RE: AceT #301174

Also CM said that Anavex have pre-clinical information for undisclosed disease, which is no surprise.

We may not be aware of all pre-clinical work Anavex have done in which diseases, but it stands to reason it is a disease that Anavex or researches testing A2-73 have published pre-clinical data on.

We know that it is not:
Infantile Spasm
Fragile X
Angelman's

The most likely is an Autism Spectrum Disorder and more specifically fragile X-associated tremor ataxia syndrome (FXTAS).

We'll see.