Category Archives: breakthrough therapy

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 , , , , , , , | 8 Comments

BTD-1…..GO!…..Musing 3

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

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

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