Tag Archives: Rett syndrome.org

Hold the Presses-Trofinetide in the News!

Posted on March 27, 2019 by melelllan

The first publication of the Pediatric Trofinetide trial has hit the proverbial newsstand! Neurology.org has published “Double-blind, randomized, placebo-controlled study of troļ¬netide in pediatric Rett syndrome” ahead of print. So, it’s hotter than “hot off the press.” So, quick cut … Continue reading →

Posted in Acadia, anxiety in Rett syndrome, Ataxia, Breathing issues in Rett syndrome, Communication, cure, Dr. Walter Kaufmann, hand clasping in Rett syndrome, Intrepid 2566, margaret brimble, Neuren Pharmaceuticals, Neurology.org, NNZ-2566, Orpan Drug Status, Rett Research, Rett Syndrome, Rett syndrome Awareness, rettsyndrome.org, screaming in rett syndrome, seizures in Rett syndrome, Texas Children's Hospital, Trail to a Texas Trial, Trofinetide, Trofinetide Pediatric trial, Uncategorized | Tagged , , , , , , , , , , , , , , , , , , , , | Leave a comment

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

Sarizotan Trial #4: Almost There!!

Posted on December 29, 2018 by melelllan

As we head into the new year, I just wanted to give you all an update on the Sarizotan trial- STARS- for daytime apnea (breath-holding). I’m pleased to announce that Newron only needs 8 (or less) more participants to complete … Continue reading →

Posted in Breathing issues in Rett syndrome, cure, hope, Newron, Rett Research, Rett Syndrome, Rett syndrome Awareness, rettland.org, rettsyndrome.org, sarizotan, Trail to a Texas Trial | 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

It’s Official!

Posted on February 16, 2015 by melelllan

This morning Neuren released a statement in regards to Orphan Drug Status for Trofinetide (NNZ 2566) See their announcement here. They’ve also come out with an awesome video about the science behind Trofinetide. Take a look! Great graphics and pretty … Continue reading →

Posted in FDA, Neuren Pharmaceuticals, NNZ-2566, Orpan Drug Status, Trofinetide | Tagged , , , , , , | Leave a comment