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Tag Archives: trofinetide pediatric trial results
Following the Yellow Brick Road!
I have been quiet of late, a lot go on in the Rettland we inhabit. A bunch of common stuff for Rett syndrome came up, doctor appts and training Katelin’s service pup, Flynn, has taken up a LOT of my … Continue reading
Let’s Break it Down-5 (Neuren Aims, Shoots, Scores! Slam Dunk!)
Yes, we have finally reached the last part of the Pediatric Trial Results. Hooray. It was quite a challenge for me and hopefully I made it understandable for everyone. I want to thank those people who explained things to me … Continue reading
Posted in cure, hope, margaret brimble, Neuren Pharmaceuticals, Rett Research, Rett Syndrome, Trail to a Texas Trial, Trofinetide, Trofinetide Pediatric trial, Uncategorized
Tagged margaret brimble, Natural History Study, Neuren, Trail to a texas Trial, trial results, Trofinetide, trofinetide pediatric trial results
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Let’s Break it Down-4
Well! If the last section was a stormy sea of up and down graphs and whirlpools of numbers, I think we’re in for some smoother sailing now! In the last post, three of the five measurements used in this trial … Continue reading
Posted in anxiety in Rett syndrome, Communication, cure, Dr. Walter Kaufmann, hand clasping in Rett syndrome, Neuren Pharmaceuticals, Rett Research, Rett Syndrome, Talking, Trail to a Texas Trial, Trofinetide, Trofinetide Pediatric trial
Tagged Dr. Walter Kaufmann, Neuren Pharmaceuticals, Trail to a texas Trial, Trofinetide, trofinetide pediatric trial results, walking and rett syndrome
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Let’s Break it Down-3 (Tipping the Scales)
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
Let’s Break it Down-2
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
Let’s Break it Down-1
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
…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