Category Archives: orphan drug designation

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,, Trail to a Texas Trial, Trofinetide, Trofinetide Pediatric trial, Trofinetide Phase III, Uncategorized | Tagged , , , , , , , | 20 Comments