As Rett syndrome Awareness Month comes to a close, I want to bring to attention two other disorders… Fragile X and Traumatic Brain Injury. What do they have to do with Rett syndrome? … Trofinetide and a chance.
In the last 30 days, Neuren has announced three major achievements: the completion of the Fragile X study; IRSF’s commitment to help fund additional trials with Trofinetide; completion of enrollment for the Intrepid study (a military funded trial of Trofinetide for TBI).
I can’t help but feel the electricity of expectation. Some parents of the Fragile X study have noticed significant improvements and I’ve seen comments regarding a lower mortality rate than expected in the TBI trial.
I feel like when you stay up all night and wish upon the stars and then off in the distance a tiny spark of light begins the dawn… And the color streams of a new day’s dawn splay out across the landscape; a promise of the coming sun. And you hold your breath as you wait for the sun to break the horizon. You know it’s coming; you know it will be glorious, this new day.
The completion of those two trials and the dedicated funds for the next steps with Trofinetide in Rett syndrome are color streams, and they are multiplying as Neuren keeps on, step by step, down the road. And when the streams have all splayed out, with only the horizon to be set ablaze, I think all of us will let out a collective sigh of wonder as the FDA places the sun in the sky and gives us all a brand new day.
TBI enrollment completion
Fragile X trial completion