Trofinetide (NNZ 2566) increases the amount of connections in your brain that control thought and movement, more connections are good.
People born with Rett syndrome have fewer synaptic connections. They also have overactive microglia, which are responsible for the maintenance of these “connective structures”.
What we hope to change
Trofinetide does 2 things:
- Helps form new spines
- Quietens the gardener (microglia)
More spines mean more synaptic connections.
Quietening the gardner (microglia) means that fewer spines are pruned which means even more synaptic connections.
Who can take it?
Trofinetide is an oral drug, there is no specific age or sex requirement. We believe that it will be available for boys and girls, regardless of their age.
However, drugs require clinical trials before they are accepted for use, and there are so few boys that it’s incredibly difficult to run an effective trial. That doesn’t mean that boys won’t be able to access it, it just means that it will take a different pathway through the FDA. The pathway will be dictated by the success of the girls’ trial.
Thank you to DR. Steve Kaminsky for everything he does.