Category Archives: margaret brimble

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

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

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 #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

Neuren Gets the Green Light!

Well, I was NOT one of the first to get to social media today with the fantabulous news that Neuren has got the go ahead to do a Phase III pivotal trial with Trofinetide. I apologize, it was a day … Continue reading

Posted in cure, FDA, hope, margaret brimble, Neuren, Neuren Pharmaceuticals, Rett Research, Rett Syndrome, Rett syndrome Awareness, rettsyndrome.org, Trail to a Texas Trial, Trofinetide, Trofinetide Phase III, Uncategorized | Tagged , , , , , , , | 4 Comments

Let’s Break it Down-5 (Neuren Aims, Shoots, Scores! Slam Dunk!)

  Yes, we have finally reached the last part of the Pediatric Trial Results. Hooray. It was quite a challenge for me and hopefully I madeĀ it understandable for everyone. I want to thank those people who explained things to me … Continue reading

Posted in cure, hope, margaret brimble, Neuren Pharmaceuticals, Rett Research, Rett Syndrome, Trail to a Texas Trial, Trofinetide, Trofinetide Pediatric trial, Uncategorized | Tagged , , , , , , | 2 Comments