Category Archives: cure

Lavender and Lilac

Allow me to introduce you to the code names of the Trofinetide Phase III! Makes me think of the time I was in the VP’s motorcade and we all had code names, lol. First up we have Lavender- a 12 … Continue reading

Posted in Acadia, cure, Neuren Pharmaceuticals, Rett Research, Rett Syndrome, Trail to a Texas Trial, Trofinetide, Trofinetide Phase III | Tagged , , , , , | 1 Comment

Rett Syndrome is Such a Trial… #1 Anavex 2-73

…and 2019 looks to change that up a bit- Rett Syndrome, so many trials! According to the most recent GP2C newsletter (GirlPower2Cure)there will be at least 7 trials this year, currently in various stages. This is SO exciting! What does … Continue reading

Posted in Anavex, Anavex 2-73, cure, Dr. Walter Kaufmann, FDA, gp2c, hope, Orpan Drug Status, Rett Research, Rett Syndrome, Rett syndrome conference 2016, Rettland Foundation, rettland.org, rettsyndrome.org, Trail to a Texas Trial, Uncategorized | Tagged , , , , , , , , , , | 3 Comments

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

In the Spotlight!- Dame Margaret Brimble

Soon the Phase III Pivotal trial for Trofinetide will begin. I have a personal goal of full recruitment within three months. That doesn’t mean everyone has started, but that all the spots are ready to be filled. WE CAN MAKE … Continue reading

Posted in cure, Dame Margaret Brimble, hope, Neuren Pharmaceuticals, nnz 2566, NNZ-2566 trial, Rett Research, Rett Syndrome, Rett syndrome Awareness, Trail to a Texas Trial, Trofinetide, Trofinetide Phase III, Uncategorized | Tagged , , , , , , , , , , , , , , , , , , , | Leave a comment

Up To The Mountain-again.

I struggled to come up with a title for this post and I struggled in deciding whether to write it or not. But, I challenged myself to be honest with both myself and others when I began this blog and … Continue reading

Posted in Acadia, anxiety in Rett syndrome, Communication, cure, Neuren Pharmaceuticals, Phone, Rett Research, Rett Syndrome, rettland.org, rettsyndrome.org, Talking, Trail to a Texas Trial, Trofinetide, Trofinetide Phase III | 6 Comments

All is Not Quiet on the Trofinetide Front #2; A Balancing Act

* Warning* Looooong post! If you aren’t interested in financial aspects and just want an update on logistics that have affected the timeline, skip to the second half- there’s also a link to a new update from Acadia, the pharmaceutical … Continue reading

Posted in Acadia, cure, FDA, Neuren Pharmaceuticals, Orpan Drug Status, orphan drug designation, Rett Research, Rett Syndrome, rettland.org, rettsyndrome.org, TBI, Trail to a Texas Trial, Trofinetide, Trofinetide Pediatric trial, Trofinetide Phase III | Tagged , , , , , , , , , , , , , , , , , , , | 7 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