Category Archives: NNZ-2566

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 , , , , , , , | 17 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

A Call to Arms!

We have yet another chance to influence the course of Rett syndrome research! Paige Nues, Director of Family Empowerment, sent out an email…In part it says: As the result of meetings just held in Washington DC, many of our elected … Continue reading

Posted in Congress, Intrepid 2566, NNZ-2566, Peer Reviewed Medical Research Program, rettsyndrome.org, TBI | Tagged , , , , , , , , , , , , , , , , , | 2 Comments