Category Archives: Trofinetide Phase III

A Little Trip, A LOT of Hope!

A few weeks ago, Katelin and I were invited to go to Acadia! On a whim, I googled “Acadia” to see what might popup. First up, beautiful Acadia National Park. Definitely would like to go there! Actually, I can’t believe … Continue reading

Posted in Acadia, cure, FDA, fund-raising for rett syndrome, hope, Neuren, Neuren Pharmaceuticals, Rett Research, Rett Syndrome, Rett syndrome Awareness, rettland.org, Trail to a Texas Trial, Trofinetide, Trofinetide Pediatric trial, Trofinetide Phase III, Uncategorized | Tagged , , , , , , , , , , , , | 2 Comments

Taking a Break

I would not normally post anything political on this blog, however I am taking a break from this blog for a bit. The current political crisis in my country is demanding my attention. In many ways, Katie’s journey will be … Continue reading

Posted in baseball, bone deformity in Rett syndrome, kyphosis, Neuren Pharmaceuticals, Rett Syndrome, Rett syndrome Awareness, Trail to a Texas Trial, Trofinetide, Trofinetide Phase III | Tagged , , , | 2 Comments

Right to Try-Walking the Tightrope

Yesterday, the president, signed the Right to Try Bill into law. But, what does that really mean for the majority of patients suffering from TERMINAL illness? I’ll try to break it down as best as possible from what I know … Continue reading

Posted in Anavex, cure, FDA, fund-raising for rett syndrome, hope, Rett Research, Right to Try Law, Trofinetide, Trofinetide Phase III, white house | Tagged , , , , | 3 Comments

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

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

What do Halloween and Rett syndrome have in Common? Superheroes.

Today is the last day of October. Another year gone by from one Awareness Month to the next and still no treatment. But this is not a humdrum post BECAUSE treatment is now within sight. Each year in October superhero … Continue reading

Posted in Anavex, Anavex 2-73, boys with Rett syndrome, cure, fund-raising for rett syndrome, Neuren Pharmaceuticals, Newron, Rett Research, Rett Syndrome, Rett syndrome Awareness, sarizotan, Trofinetide, Trofinetide Phase III, Uncategorized | 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