how many other neuro developmental diseases may be included in this mold? The genes are within the code, but not properly expressed by chromatin?
All of what I speculate on here is just that, speculation.
But it's based upon newly-discovered science, where the Anavex sigma-1 receptor activators ("agonists") not only facilitate a) proper protein folding, b) various cellular homeostatic processes, and c) autophagy, but also, now, d) chromatin modulation during gene expression.
In fact, as studies will eventually determine, chromatin function modulation by Anavex molecules may turn out to be their most significant application, even greater than central nervous system (CNS) disease and aging prophylaxis.
As with fragile-X syndrome, a large number of diseases and conditions are caused by gene expression dysfunction. The genes are in the DNA, in the chromosomes, and often even get properly expressed in younger periods of life. But at various times, for various reasons, the chromatin wrapped around the compressed coils of DNA in the chromosomes fails to fully unwrap, or otherwise interferes with gene expression.
So, my speculation is that, someday, blarcamesine (or Anavex 3-71) will become a universally administered prophylactic therapy to everyone, even to mothers prenatally, to minimize birth defects resulting from dysfunctional gene expression.
I needn't prompt anyone to ponder the multitude of significances of such a discovery and implementation. We'll be watching for any of this. Might it prompt a rephrasing of a comment made some time ago by an Anavex principal, about the company's drugs, "Like an iceberg?"
That might have been an understatement. Since then, with all we know about Anavex sigma-1 receptor activation, this, now is the applicable statement: "Applications like the size of a continental glacier!"
Except for effective doses of blarcamesine, nothing minute about Anavex.