Monthly Archives: March 2017

Let’s Break it Down-3 (Tipping the Scales)

Posted on March 31, 2017 by melelllan

So, here we go. I struggled with this section because of the graphs and numbers. There may be many of you who go…”What’s the big deal, so easy to understand!” Me, to say I’m good at math would be stretch … Continue reading →

Posted in Neuren Pharmaceuticals, Trail to a Texas Trial, Trofinetide, Trofinetide Pediatric trial, ucla statistics dept., Uncategorized | Tagged , , , , , | 2 Comments

Let’s Break it Down-2

Posted on March 28, 2017 by melelllan

So, the last post gave a rundown on the summary, now we’ll get into the nitty gritty! Let’s break down the next section: *disclaimer: these are my thoughts only, unless otherwise noted. The Phase 2 trial was conducted at 12 … Continue reading →

Posted in Neuren Pharmaceuticals, Rett Research, Rett Syndrome, Trail to a Texas Trial, Trofinetide, Trofinetide Pediatric trial, Uncategorized | Tagged , , , , , | 3 Comments

Let’s Break it Down-1

Posted on March 24, 2017 by melelllan

  So, the Rett Community has had a couple of days to digest the BIG news-Trofinetide showed clinically significant improvement in the Pediatric Trial. Then we read the announcement, full of graphs and numbers and a lot of us went… … Continue reading →

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

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

Posted on March 21, 2017 by melelllan

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

Posted on March 21, 2017 by melelllan

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-?Hours: Musing 2-The Stuff of Dreams

Posted on March 21, 2017 by melelllan

So, the results didn’t come out today. Bummer, that. Well, then, it’s just another sleep and a wakeup give or take. So we all get to sleep on it. I’m not worried about tomorrow; I’m certain that, no matter what … Continue reading →

Posted in boys with Rett syndrome, cure, hope, Neuren Pharmaceuticals, Rett Research, Rett Syndrome, Trail to a Texas Trial, Trofinetide Pediatric trial, Uncategorized | Tagged , , , , , | 1 Comment

T- ?hours:Musing 1- Hands are Clasped

Posted on March 20, 2017 by melelllan

Hand clasping is the signature sign of Rett Syndrome. It prevents most girls from using their hands. Katie is very, very fortunate to have pretty good hand use, incredible hand use if you take Rett syndrome into consideration. This morning … Continue reading →

Posted in hand clasping in Rett syndrome, Neuren Pharmaceuticals, Rett Syndrome, Trofinetide, Trofinetide Pediatric trial, Uncategorized | Tagged , , , | Leave a comment

Happy St. Patrick’s Day…a Lucky Day for… EVERYONE!

Posted on March 17, 2017 by melelllan

The luck of the Irish is spreading for the Rett community. Oh, no, it’s coming from Australia this year. Neuren Pharmaceuticals Ltd- * Requests trading halt pending announcement regarding Neuren’s phase 2 clinical trial of Trofinetide in Rett Syndrome. Yes … Continue reading →

Posted in Neuren Pharmaceuticals, Trofinetide, Trofinetide Pediatric trial, Uncategorized | Tagged , , , , , | 2 Comments

T-? Crunching the Numbers

Posted on March 16, 2017 by melelllan

  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

T-? Closing in on the 2nd Finish Line

Posted on March 11, 2017 by melelllan

I’m musing today, as I think about the three major trials related to Trofinetide, that the trials are like the Triple Crown. Now, I don’t know much about horse racing (though the Rett community does have its very own race horse, … Continue reading →

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