Thanks Tony,
Yes, I have read the article but re-read following Professor Snape's presentation. The scientific background helps in understanding the mechanism of action of NNZ-2566.
The big question for NNZ-2566 was whether the results in mouse trials for Rett syndrome would be replicated in the Phase II trials. Of course we will not know until next year yet an extraordinary window opened up through which we caught a glimpse of patients undergoing medication.
I was aware of the comments in Facebook back in July when I wrote;
"Parents will observe the behavioural impact of the treatment; hand wringing, verbalisation, rigidity of body movement, calmness and alertness; seizures and breathing patterns."
The observations by parents of their Rett girls and women, however, were intensely personal. I felt privileged to hear their stories and equally reluctant to intrude in any way because I have a vested financial interest in the success of NNZ-2566.
Due to the speculation surrounding the release of social media comments, the window has now probably closed. Nonetheless, what we saw was incredibly exciting. Some parents saw in their Rett girls and women what Professor Snape saw in mouse trials: changes in behaviour following medication.
What is true in Rett will almost certainly be true in Fragile X. What is true in Rett and Fragile X points towards a kind of universal drug for all "CNS conditions at the level of disease causation". So we are talking about Alzheimers disease, Parkinsons disease, autism, TBI and dementia. A universal drug in an unmet market.
Wayne.
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