Category Archives: rettsyndrome.org

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

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

A Month of Awareness

  October is Rett syndrome Awareness Month. This means of course to make OTHER people aware of Rett syndrome; for us parents, siblings, grandparents, caregivers the whole YEAR is awareness month.   So, how to make people aware? There are … Continue reading

Posted in Army of us, cure, fund-raising for rett syndrome, Rett Research, Rett Syndrome, rettsyndrome.org, Special needs siblings, Trail to a Texas Trial | Tagged , , , | 5 Comments

…And Slam Dunk Pulls Ahead of the Pack to Win by a Mile!!!

It wasn’t even a photo finish! Neuren’s trial was a double-blind, randomized, placebo controlled study that tested three doses of trofinetide compared with placebo in 82 subjects.  The highest dose of trofinetide achieved statistically significant clinical benefit compared with placebo … Continue reading

Posted in Neuren Pharmaceuticals, Rett Research, Rett Syndrome, rettland.org, rettsyndrome.org, Trofinetide, Trofinetide Pediatric trial, Uncategorized | Tagged , , | 2 Comments

T-?Minutes: Musing 3-Down to the Wire

Well, we’re down to the wire now. This race of results is coming to an end. What a wild ride the last few days have been for the Rett community. Time to stand up and peer down the final stretch. … Continue reading

Posted in Neuren Pharmaceuticals, Rett Research, Rett Syndrome, rettland.org, rettsyndrome.org, Trail to a Texas Trial, Trofinetide Pediatric trial, Uncategorized | Tagged , , , , , | 2 Comments

T-? Crunching the Numbers

  There are 31 days in March. That means at noon today, we entered the second half. Which, in turn, means there are less than 15 days until we will know the results of the Trofinetide Pediatric Trial. 360 hours. … Continue reading

Posted in boys with Rett syndrome, cure, hope, Neuren Pharmaceuticals, Rett Research, Rett Syndrome, rettsyndrome.org, Trail to a Texas Trial, Trofinetide, Trofinetide Pediatric trial | Tagged , , , , , , , | Leave a comment

Too Much of a Good Thing…Makes me Way Behind!

SO much is going on in the world of Rett syndrome, that I have fallen woefully behind in keeping up. To my defense a lot has been going on in my little world that needed attention, BUT as a State … Continue reading

Posted in boys with Rett syndrome, MecP2 duplication, Rett Research, Rett Syndrome, Rett syndrome Conference 2018, rettsyndrome.org, Trail to a Texas Trial | Tagged , , , , , , | Leave a comment