Category Archives: boys with Rett syndrome

Close is Too Late for Too Many

As we approach the start of the Trofinetide trial, I know that it is going to go by in a blink.  And, there are other trials in different stages of development, each holding some parent’s hope for their child- improvement … Continue reading

Posted in Acadia, Anavex, Avexis, boys with Rett syndrome, cure, Death in Rett syndrome, grief, Neuren Pharmaceuticals, Rett Research, Rett Syndrome, Rett syndrome Awareness, tracy chapman, Trail to a Texas Trial, Trofinetide Phase III | Leave a comment

One Moment in Time…

To be honest, I had to wait over a day to try and write this post. And still, I start crying; I ramble; I’ve looked at what I’ve written and while to a special needs’ parent it may look coherent, … Continue reading

Posted in America's Got Talent, boys with Rett syndrome, Communication, hope, Kodi Lee, Music, Rett Syndrome, Rett syndrome Awareness, screaming in rett syndrome, seizures in Rett syndrome, Special needs siblings, Trail to a Texas Trial, Trofinetide | Tagged , , , , , , , , , , , | 3 Comments

All is Not Quiet on the Trofinetide Front #1 A Chess Game

It’s been very quiet in the public eye in regard to Trofinetide. We all are in a holding pattern as we wait for Phase III to begin, but BEHIND the scenes things are progressing strategically and clearly, like a chess … Continue reading

Posted in Acadia, anxiety in Rett syndrome, Ataxia, boys with Rett syndrome, breakthrough therapy, Communication, cure, FDA, Fragile X, hope, margaret brimble, Neuren Pharmaceuticals, Rett Research, Rett Syndrome, rettland.org, rettsyndrome.org, Trail to a Texas Trial, Trofinetide, Trofinetide Pediatric trial, Trofinetide Phase III, Trofinitide, Uncategorized | Tagged , , , , , , , , , , , , , , , , , , , , , | Leave a comment

Winding Down

Rett syndrome Awareness #30 October is winding down and we’ve almost made it through the month. As I sit here and write this, I know there are families who didn’t make it through, they lost their children in the days … Continue reading

Posted in boys with Rett syndrome, cure, fund-raising for rett syndrome, Huda Zoghbi, margaret brimble, Neuren Pharmaceuticals, Rett Research, Rett Syndrome, Rett syndrome Awareness, rettland.org, rettsyndrome.org, RSRT, Trail to a Texas Trial, Uncategorized | Tagged , , , , , , , , , , , | Leave a comment

Trofinetide #3

And on we go! So far I’ve discussed what Trofinetide is, how it came about and what it does. Great, but what are we talking for a timeline, right? Who holds the strings? Just what the heck are the steps … Continue reading

Posted in boys with Rett syndrome, breakthrough therapy, FDA, hope, margaret brimble, Neuren Pharmaceuticals, NNZ-2566, Orpan Drug Status, orphan drug designation, Rett Syndrome, rettland.org, Trail to a Texas Trial, Trofinetide, Trofinetide Pediatric trial, Trofinetide Phase III, Uncategorized | Tagged , , , , , , , | 20 Comments

Trofinetide #2

So, here we go- Why was Rett syndrome chosen for Trofinetide trials. Contrary to what most people think, Trofinetide was not developed for Rett syndrome. A quick google search reveals that it was first developed for stroke, but the focus … Continue reading

Posted in boys with Rett syndrome, cure, Fragile X, hope, Intrepid 2566, Neuren Pharmaceuticals, NNZ-2566, Rett syndrome Awareness, TBI, Trail to a Texas Trial | Tagged , , , , , , | Leave a comment

Trofinetide #1

So, we are narrowing in on the start of Phase III of Trofinetide. It’s a word that causes caution, skepticism, hope, giddiness and a myriad of other emotions. Even though I am completely convinced of it’s effectiveness, how it affects … Continue reading

Posted in boys with Rett syndrome, Communication, cure, Fragile X, hope, margaret brimble, Neuren Pharmaceuticals, NNZ-2566, Rett Research, Rett Syndrome, Talking, Texas Children's Hospital, Trail to a Texas Trial, Trofinetide | Tagged , , , , , , | Leave a comment