Category Archives: Rett Syndrome

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

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

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

Body Bound by a Brain #4

A roller coaster twists and turns and bends. It can take your breath away. So does Rett syndrome, in a whole other, non-exhilarating, way. Rett syndrome twists and turns and bends our children’s bodies, often taking their breath away.   … Continue reading

Posted in bone deformity in Rett syndrome, kyphosis, Rett Research, Rett Syndrome, Rett syndrome Awareness, scoliosis, Trail to a Texas Trial, Uncategorized | Tagged , , , , , | 1 Comment

A Body Bound by a Brain #3

Oh, don’t us parents, all of us, just get so excited when our babies pass milestones- the first smile, the first laugh, the first time rolling over, the first step, the first word. The first word. That brilliant first word, … Continue reading

Posted in anxiety in Rett syndrome, boys with Rett syndrome, Communication, cure, Neuren Pharmaceuticals, NNZ-2566 trial, Phone, Rett Research, Rett Syndrome, Rett syndrome Awareness, rettland.org, rettsyndrome.org, screaming in rett syndrome, Talking, Trail to a Texas Trial, Trofinetide, Trofinetide Pediatric trial, Uncategorized | Tagged , , , , , , , , | 1 Comment