Tag Archives: Phase III trofinetide

All is Not Quiet on the Trofinetide Front #2; A Balancing Act

Posted on January 8, 2019 by melelllan

* Warning* Looooong post! If you aren’t interested in financial aspects and just want an update on logistics that have affected the timeline, skip to the second half- there’s also a link to a new update from Acadia, the pharmaceutical … Continue reading →

Posted in Acadia, cure, FDA, Neuren Pharmaceuticals, Orpan Drug Status, orphan drug designation, Rett Research, Rett Syndrome, rettland.org, rettsyndrome.org, TBI, Trail to a Texas Trial, Trofinetide, Trofinetide Pediatric trial, Trofinetide Phase III | Tagged , , , , , , , , , , , , , , , , , , , | 7 Comments

Taking a Break

Posted on June 28, 2018 by melelllan

I would not normally post anything political on this blog, however I am taking a break from this blog for a bit. The current political crisis in my country is demanding my attention. In many ways, Katie’s journey will be … Continue reading →

Posted in baseball, bone deformity in Rett syndrome, kyphosis, Neuren Pharmaceuticals, Rett Syndrome, Rett syndrome Awareness, Trail to a Texas Trial, Trofinetide, Trofinetide Phase III | Tagged , , , | 2 Comments

What do Halloween and Rett syndrome have in Common? Superheroes.

Posted on October 31, 2017 by melelllan

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

Neuren Gets the Green Light!

Posted on October 13, 2017 by melelllan

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