Yes, the drugs Neuren have developed are doing the job of something that is missing in the brains of Rett’s sufferers due to a genetic omission in their DNA, which means that because of this, the RNA strand that would normally have the necessary protein synthesis on is missing and therefore, if they stop taking the drug, the IGF will become unregulated again and their brain function will deteriorate.
At least that is how I understand it from all the presentations etc from the company, and a bit of HSC biology and knowledge about anti sense drugs.
There are medical professionals posting here that might be able to add to this topic or explain it better than me I guess, so any further insight is welcome by me.
The only caveat I would apply is that one day something better might come along via the genetic manipulation route that would make Neuren’s drugs obsolete. Cheers.
Yes, the drugs Neuren have developed are doing the job of...
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