Anavex Life Sciences Corp (AVXL)

[nidan7500]

"Targeting the ultimate cause of the disease would be expected to have better outcomes than addressing its many downstream effects."
Lee adds, "The approach described in our work takes advantage of the fact that every patient carries a cure within her own cells. But that cure is locked up by a life-long process called X-chromosome inactivation. Our goal has been to unlock the inactive X and restore expression of the good gene copy."
Females carry two copies of the X chromosome, but within each cell only one copy is active, the other being silenced by an RNA molecule called Xist. Which copy remains active in which cell is randomly determined during embryonic development. While some X-linked disorders only produce symptoms in males, who carry a single X chromosome, females can be affected if a mutation is in a dominant gene on the active X chromosome.

https://www.sciencedaily.com/releases/2018/01/180104131609.htm

Great news, an awareness of patient born-self healing when treated and biotech product development process changes occurring simultaneously. FDA type trials process rules are unable to address the kind of change that new science needs.(merging S/W models and precision medicine based disciplines). Trying to produce formal trials results while this chaos is afoot is tough to do. W/S and BP are going to have to "wing it" for a while. "They cannot handle the truth".