Category Archives: NNZ-2566

Intrepid-2566

Posted on March 30, 2023 by melelllan

This blog is going to be from memory, though I think I’ll get it right. Intrepid-2566, such a grand undertaking-to save the soldiers that suffered TBI. One might think being a veteran this one is near and dear to my … Continue reading →

Posted in Daybue, Intrepid 2566, Neuren Pharmaceuticals, NNZ-2566, TBI, Trofinetide | Tagged | Leave a comment

Where do we go From Here? Part I

Posted on August 10, 2022 by melelllan

Last week, the International Rett syndrome Foundation (IRSF) held a webinar with Dr. Dominique Pichard, CSO, IRSF and Mr. James E. Valentine, Esq., HPM aptly named, “What Comes Next? The FDA Regulatory Process from NDA to FDA decision and beyond.” … Continue reading →

Posted in Acadia, clinicaltrials.gov, Dame Margaret Brimble, FDA, International Rett syndrome Foundation, Intrepid 2566, LAVENDER TRIAL, Neuren Pharmaceuticals, NNZ-2566, NNZ-2566 trial, orphan drug designation, Rett Research, Rett Syndrome, rettsyndrome.org, TBI, Trail to a Texas Trial, Trofinetide Pediatric trial, Trofinetide Phase III | Tagged , , , , , , , | Leave a comment

Trofinitide: A Parental Case Study

Posted on June 21, 2022 by melelllan

As the trail to Trofinitide is coming to its conclusion- the long awaited will they/won’t they approve, I want to revisit Katelin’s experience during the trial, when parents weren’t sworn to silence. It’s been a long, nearly nine years and … Continue reading →

Posted in Acadia, Neuren Pharmaceuticals, NNZ-2566, NNZ-2566 trial, Trail to a Texas Trial, Trofinetide | Tagged , , , , , , , | 3 Comments

Trofinetide Series Part 3-LAVENDER- Arms and Interventions; Outcome Measures-Primary AKA a Leap Toward Proving Efficacy

Posted on February 10, 2020 by melelllan

Deep breath everybody. This is starting to get into the nitty gritty of what the hopes are for Trofinetide and how success will be measured. This can get complicated and I will try my very best to make it easy … Continue reading →

Posted in Acadia, anxiety in Rett syndrome, boys with Rett syndrome, clinicaltrials.gov, Dame Margaret Brimble, FDA, Neuren Pharmaceuticals, NNZ-2566, NNZ-2566 trial, Rett Research, Rett Syndrome, screaming in rett syndrome, Trofinetide, Trofinetide Pediatric trial, Trofinetide Phase III, Uncategorized | Tagged , , , , , , , | Leave a comment

Our Unconquerable Souls

Posted on January 29, 2020 by melelllan

Nearly 70 years ago, Andreas RettĀ  observed two girls in his office with similar symptoms and thus began the known journey of Rett syndrome. In 1966 several German publications recognized his work. However, it wasn’t until 1983 that Dr. Bengt … Continue reading →

Posted in Acadia, Army of us, boys with Rett syndrome, clinicaltrials.gov, Dame Margaret Brimble, Huda Zoghbi, Neuren Pharmaceuticals, NNZ-2566, NNZ-2566 trial, Rett syndrome Awareness, screaming in rett syndrome, seizures in Rett syndrome, Trail to a Texas Trial, Trofinetide, Trofinetide Pediatric trial, Trofinetide Phase III | Tagged , , , , , , , | 2 Comments

Trofinetide Series-Part I: Words you Need to Know

Posted on January 17, 2020 by melelllan

So, over the course of the next few weeks, I’m going to be doing a series of posts to catch you all up to speed on the Trofinetide trial, which for many of you seems to be going at a … Continue reading →

Posted in Acadia, boys with Rett syndrome, Dame Margaret Brimble, FDA, Intrepid 2566, Neuren Pharmaceuticals, NNZ-2566, NNZ-2566 trial, Orpan Drug Status, Rett Research, Rett Syndrome, TBI, Trail to a Texas Trial, Trofinetide, Trofinetide Pediatric trial, Trofinetide Phase III | Tagged , , , , , , , | 1 Comment

A Day with Acadia- Part 1: Awareness

Posted on October 30, 2019 by melelllan

This is my last post of Rett syndrome Awareness month. I had hoped to do more, but Rett syndrome made sure a good amount of awareness was happening right at home, but this was going to be the last either … Continue reading →

Posted in Acadia, Anavex, Anavex 2-73, Communication, Dame Margaret Brimble, fund-raising for rett syndrome, gp2c, hope, ketamine, kyphosis, Neuren, Neuren Pharmaceuticals, Newron, NNZ-2566, NNZ-2566 trial, Remarkable Rett-a podcast series, Rett Research, Rett Syndrome, Rett syndrome Awareness, rettsyndrome.org, RSRT, sarizotan, service dog, Talking, Trail to a Texas Trial, Trofinetide, Trofinetide Phase III | Leave a comment

A Mother Meets a Child She Will Give New Life To

Posted on August 29, 2019 by melelllan

It is no secret that I have high expectations for Trofinetide, I fully expect Katelin will speak; I fully expect her to be able to read and write; I expect that she will be calm, well she is Irish, so … Continue reading →

Posted in Acadia, ACS Medicinal Chemistry Division, American Chemical Society, Dame Margaret Brimble, hope, Mark Brimble, Neuren Pharmaceuticals, NNZ-2566, NNZ-2566 trial, Rett Syndrome, TBI, Trail to a Texas Trial, Trofinetide, Trofinetide Pediatric trial, Trofinetide Phase III | Tagged , , , , , , , , , , | 7 Comments

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

Trofinetide #3

Posted on January 19, 2018 by melelllan

And on we go! So far I’ve discussed what Trofinetide is, how it came about and what it does. Great, but what are we talking for a timeline, right? Who holds the strings? Just what the heck are the steps … Continue reading →

Posted in boys with Rett syndrome, breakthrough therapy, FDA, hope, margaret brimble, Neuren Pharmaceuticals, NNZ-2566, Orpan Drug Status, orphan drug designation, Rett Syndrome, rettland.org, Trail to a Texas Trial, Trofinetide, Trofinetide Pediatric trial, Trofinetide Phase III, Uncategorized | Tagged , , , , , , , | 20 Comments