Category Archives: breakthrough therapy

All is Not Quiet on the Trofinetide Front #1 A Chess Game

Posted on January 6, 2019 by melelllan

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

Trofinetide #3

Posted on January 19, 2018 by melelllan

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

BTD-1…..GO!…..Musing 3

Posted on February 27, 2015 by melelllan

Today, as we reach the 60 day limit from application to hopefully an announcement. I have a simple post. Our wishes have been READY…. . . . . . . . . We’ve been SET for a long time…. . … Continue reading →

Posted in breakthrough therapy, FDA, Neuren, NNZ-2566, Rett Syndrome, rettsyndrome.org, Trail to a Texas Trial, Trofinetide | Tagged , , , , , , , | Leave a comment

BTD-2……..Set…… Musing 2

Posted on February 26, 2015 by melelllan

I am a researching junkie. True. Difficult to believe, I know. 😉 I like being “set”. I like knowing what I’m getting in for, whether it’s figuring out my daughter had Rett syndrome before any doctor would say “Your daughter … Continue reading →

Posted in breakthrough therapy, cure, FDA, Neuren Pharmaceuticals, Rett Syndrome, rettsyndrome.org, Trail to a Texas Trial | Tagged , , , | 2 Comments

Please, Please, Please, Please

Posted on February 24, 2015 by melelllan

One “please” for evey day left. Rett parents say so many “pleases” that never get answered. Please, don’t let that MecP2 test come out positive. Please, don’t let my baby lose her words. Please, let me hear my baby say … Continue reading →

Posted in breakthrough therapy, FDA, hope, Neuren Pharmaceuticals, Rett Syndrome | Tagged , , , , | 2 Comments