" Taken together, these behavioral paradigms measure different aspects of muscular coordinaton, balance, motor learning and muscular strengths, some of the core deficits observed in Rett syndrome"
" Administra,on of ANAVEX 2-73 results in both significant and dose related improvements in an array of behavioral paradigms in the MECP2 HET Rett syndrome disease model"
Since Rett Syndrom Foundation is financially supporting AVXL trials, it is logically expected, that RSF will encourage parents to participate in this particular trial. How many patients do we need?
The theory of restored homeostasis addresses Rett by postulating that the ER is misfolding proteins due to insufficient ATP and perhaps other molecules produced by the mitochondria supplying the ER.
By reestablishing the connection between the mitochondria and the ER the misfolded and therefore ineffective proteins will no longer be produced.
The result is the ER is now producing effective proteins. If it is this faulty connection mechanism that results in insufficient MeCP2 protein then 2-73 becomes a plausible mechanism for creating the proper amount.
Obviously there are multiple other links in the chain that leads to insufficient MeCP2 protein levels that might not be addressed by 2-73.
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