I have been quiet of late, a lot go on in the Rettland we inhabit. A bunch of common stuff for Rett syndrome came up, doctor appts and training Katelin’s service pup, Flynn, has taken up a LOT of my time. But a few BIG things have happened since last I posted. First, Katelin turned 24!! That is two times the age I was told she would live to. So take that Rett syndrome. So, that called for special times as family flew in from Massachusetts and Seattle and her old aide, Betty, came to stay for a week and both got treated!
We’ve been kept making presents and other artwork at Color Me Mine and hanging with the grandkids.
Not to mention concerts, plays, baseball and softball!!
But, I need to acknowledge tremendous news about Neuren and Trofinetide.
Neuren has secured the funding to move ahead with the Phase III trial. See their announcement-here and will be meeting with the FDA this summer ” to discuss the remaining development for trofinetide to treat Rett syndrome, including the Phase 3 trial design.”
The golden bricks are being laid out one by one now, very quickly but carefully! No tripping us up, now.
I also want to comment on an old article from Psychology Weekly that’s been making the rounds on Facebook about the reversibility of Rett syndrome and how they talk about medications that may do what activating the “good gene” would do- regrow the dendritic halo. That is the goal of Trofinetide and what it has been shown to begin to accomplish during the short trials that have occurred. So, for those that worry that gene therapy may come too late to cure your children, there really is hope in Trofinetide being able to modify the course of the disorder, as their report on the Phase II results mentions.
Believe me, I know it’s hard to be patient. It seems like FOREVER, but next year is now less than 6 months away, the new and LAST trial will be upon us soon. And, then, perhaps our children will be as in the words of Martin Luther King, Jr. “Free at last, Free at last.”