Category Archives: hope

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

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

Trofinetide #2

So, here we go- Why was Rett syndrome chosen for Trofinetide trials. Contrary to what most people think, Trofinetide was not developed for Rett syndrome. A quick google search reveals that it was first developed for stroke, but the focus … Continue reading

Posted in boys with Rett syndrome, cure, Fragile X, hope, Intrepid 2566, Neuren Pharmaceuticals, NNZ-2566, Rett syndrome Awareness, TBI, Trail to a Texas Trial | Tagged , , , , , , | Leave a comment

Trofinetide #1

So, we are narrowing in on the start of Phase III of Trofinetide. It’s a word that causes caution, skepticism, hope, giddiness and a myriad of other emotions. Even though I am completely convinced of it’s effectiveness, how it affects … Continue reading

Posted in boys with Rett syndrome, Communication, cure, Fragile X, hope, margaret brimble, Neuren Pharmaceuticals, NNZ-2566, Rett Research, Rett Syndrome, Talking, Texas Children's Hospital, Trail to a Texas Trial, Trofinetide | Tagged , , , , , , | Leave a comment

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

Christmas Wonderland

Every year for the past 18 years I’ve asked Santa for a cure for Rett syndrome. (Katie was 6 when the gene testing became available and we knew with absolute certainty she had Rett syndrome). Every year except this year. … Continue reading

Posted in Communication, hope, Neuren Pharmaceuticals, Rett Research, Rett Syndrome, Talking, Trail to a Texas Trial, Uncategorized | Tagged , , , , , , | 1 Comment