Today Neuren released its top line results of the Fragile X clinical trial with Trofinetide. Fragile X, much more prevalent than Rett syndrome, affects approx. 1 in 4000 boys and 1 in 4000-6000 girls. This study was done with boys 16-45 mimicking the Rett syndrome study, just the opposite sex.
All the following is my own opinion. I believe that it was strategic and fair principal, to do it on opposite sexes. This proves safety in both sexes in addition to success in core factors for the different syndromes- in Rett syndrome not enough connections and a huge synaptic space; in Fragile X, too many connections, and the same medication, given the same way, at the same doses proves efficiency in alleviating symptoms. I liken this to the same thyroid medication that fixes both hypo and hyper thyroidism.
I cannot even tell you how hopeful this makes me, that MAYBE with the (hopefully) successful TBI trial, the FDA will speed along approval. No matter what though, this is HUGE. And, I am so happy for all those sufferers of Fragile X and their families that, like for us Rett families, a real Hope has emerged.
To all those who have worked on Trofinetide, sponsored trials and have helped in so many ways, sleep well tonight. You have done well.
Ps: I never had a single doubt 🙂 xo