Category Archives: NNZ-2566

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

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

Epic Fail (Not)

Today was an epic fail on my part. And so I will generalize this day and trials, not just Trofinetide, but just in general. I started out with a GREAT premise…Katie would LOVE a whale watch. We were on one … Continue reading

Posted in cure, Intrepid 2566, Neuren, Neuren Pharmaceuticals, NNZ-2566, TBI, Trail to a Texas Trial, Trofinetide, Uncategorized | Leave a comment

Three more Colors of Dawn

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 … Continue reading

Posted in cure, FDA, Fragile X, fund-raising for rett syndrome, hope, Intrepid 2566, Neuren Pharmaceuticals, NNZ-2566, Rett Syndrome, TBI, Trofinetide, Uncategorized | Tagged , , , , , , , , | 3 Comments

Newsworthy News!

Today, like many other Rett parents, I got an e-mail from Neuren in regards to their Chairman’s Address to Stockholders. It was chock full of hopeful expectations. Of particular note: “Finally, I would like to make some remarks in relation … Continue reading

Posted in Ann Romney Center for Neurological Diseases, Brigham and Women's, cure, Neuren Pharmaceuticals, NNZ-2566, Rett Research, Rett Syndrome, rettland.org, TBI, Trofinetide | Tagged , , , , , , , , , , | 1 Comment