Category Archives: Ataxia

Hold the Presses-Trofinetide in the News!

The first publication of the Pediatric Trofinetide trial has hit the proverbial newsstand! Neurology.org has published “Double-blind, randomized, placebo-controlled study of troļ¬netide in pediatric Rett syndrome” ahead of print. So, it’s hotter than “hot off the press.” So, quick cut … Continue reading

Posted in Acadia, anxiety in Rett syndrome, Ataxia, Breathing issues in Rett syndrome, Communication, cure, Dr. Walter Kaufmann, hand clasping in Rett syndrome, Intrepid 2566, margaret brimble, Neuren Pharmaceuticals, Neurology.org, NNZ-2566, Orpan Drug Status, Rett Research, Rett Syndrome, Rett syndrome Awareness, rettsyndrome.org, screaming in rett syndrome, seizures in Rett syndrome, Texas Children's Hospital, Trail to a Texas Trial, Trofinetide, Trofinetide Pediatric trial, Uncategorized | Tagged , , , , , , , , , , , , , , , , , , , , | Leave a comment

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

Rett syndrome Awareness #8

I’m sure people reading this blog are going to go… what happened to the other 7??? Some have been close to posts on this blog so I’ve just posted to Facebook, but I don’t believe I’ve touched upon this particular … Continue reading

Posted in Acadia, Ataxia, cure, Fragile X, margaret brimble, Neuren Pharmaceuticals, Rett Research, Rett Syndrome, Rett syndrome Awareness, Uncategorized | Tagged , , , , , , | Leave a comment