Category Archives: NNZ-2566 trial

17th of March in September

Posted on September 2, 2022 by melelllan

Katelin was in the NNZ-2566 (Trofinetide) trial through the holidays the year she was in Texas. We were incredibly blessed. One family was having a party for Thanksgiving at the hotel we were in and shared their meal with us. … Continue reading →

Posted in Acadia, Neuren Pharmaceuticals, NNZ-2566 trial | 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

It’s the Home Stretch!

Posted on July 18, 2022 by melelllan

ACADIA has announced the submission of Trofinetide’s application to the FDA! The words we have been waiting for have finally been written. It’s a couple of weeks later than our hearts were shooting for, but better late and the best … Continue reading →

Posted in Acadia, Daffodil trial, FDA, LAVENDER TRIAL, Neuren, NNZ-2566 trial, orphan drug designation, Rett Research, Rett Syndrome, Trail to a Texas Trial, Trofinetide, Trofinetide Pediatric trial, Trofinetide Phase III, Uncategorized | 2 Comments

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 Rett  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

In the Spotlight!- Dame Margaret Brimble

Posted on March 26, 2019 by melelllan

Soon the Phase III Pivotal trial for Trofinetide will begin. I have a personal goal of full recruitment within three months. That doesn’t mean everyone has started, but that all the spots are ready to be filled. WE CAN MAKE … Continue reading →

Posted in cure, Dame Margaret Brimble, hope, Neuren Pharmaceuticals, nnz 2566, NNZ-2566 trial, Rett Research, Rett Syndrome, Rett syndrome Awareness, Trail to a Texas Trial, Trofinetide, Trofinetide Phase III, Uncategorized | Tagged , , , , , , , , , , , , , , , , , , , | Leave a comment