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kld2

09/02/17 8:32 AM

#118274 RE: F1ash #118273

The wishful thinking is hopeless. Good example here. Article on un-named company focused on protein replacement suddenly becomes Anavex. Por favor!
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Amatuer17

09/02/17 8:54 AM

#118276 RE: F1ash #118273

Thanks for the articles - the trial participation and patient availability is a big concern in many diseases as there are competing trials and patients are spread all over US - this is applicable for Rett as well.

Side question - "I have been particularly encouraged by breakthrough scientific advances that may ultimately enable therapeutics directly targeting the cause of Rett syndrome – insufficient MeCP2 protein (e.g., gene therapy, X-reactivation, protein replacement)"

If cause of Rett is insufficient MeCP2 protein - why A2-73 will be effective if it addresses homeostasis and not protein?