Category Archives: FDA

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

Here’s to a New Year and a New Hope

This will be my last post of the year!   I’m feeling particularly retrospective at this time, this year. Facebook has reminded me on an almost daily basis since November of Katelin’s participation in the Adult trial (Phase II of … Continue reading

Posted in boys with Rett syndrome, Communication, cure, FDA, hope, MecP2 duplication, Neuren Pharmaceuticals, NNZ-2566 trial, Rett Research, Rett Syndrome, rettsyndrome.org, Talking, Texas Children's Hospital, Trail to a Texas Trial, Trofinetide, Trofinetide Pediatric trial, Trofinetide Phase III | Tagged , , , , , , , , , , | 2 Comments

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

We are Nearing the True Unknown

So, we are closing in on the third and final pivotal trial of Trofinetide. It is my understanding that Neuren and the FDA are meeting in October. Sometime next year the study will begin recruiting and start. What the criteria … Continue reading

Posted in acceptance, boys with Rett syndrome, Communication, cure, FDA, hope, Neuren Pharmaceuticals, Rett Research, Rett Syndrome, Talking, Trail to a Texas Trial, Trofinetide, Uncategorized | Tagged , , , , , , | 3 Comments

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

20 Years in Oz

I saw a writing contest advertised on Facebook, the very next ad under it was something to do with the Wizard of Oz…and so I was inspired. I had hoped to get this published, but no takers. However since this … Continue reading

Posted in cure, FDA, nnz 2566, Rett Syndrome, Trail to a Texas Trial, Trofinetide, Uncategorized, Wizard of Oz | Tagged , , , , | 3 Comments

Tonight should be the Day

If┬áthe FDA is on target with their 60 days, Neuren should have been notified over the weekend; so, around 6 pm or 7 pm US EST, we should know what the FDA has said about Breakthrough Therapy Designation. Over the … Continue reading

Posted in FDA, Neuren Pharmaceuticals, Rett Syndrome | Tagged , , , , , | 5 Comments