Category Archives: LAVENDER TRIAL

IRSF Scientific Meeting, Daybue and More

Posted on June 19, 2023 by melelllan

The International Rett Syndrome Foundation (IRSF) held its Scientific Meeting on June 5-7, featuring presentations from Acadia, Taysha, Neurogene, and NeuroTec. Anavex did not present as they had no new information to share. Acadia presented LILAC data and introduced a new Real World Efficacy study called LOTUS. Unfortunately, any data presented at the meeting is not publicly available, but a summary will be provided by IRSF at a later date. Dr. Jim Youakim gave a historical presentation about Trofinetide’s development, with an emphasis on the LAVENDER study and challenges Acadia faced during the pandemic. Continue reading →

Posted in Acadia, Anavex, Daybue, IRSF, LAVENDER TRIAL, Neuren Pharmaceuticals, Rett Syndrome, Trail to a Texas Trial, Uncategorized | Tagged , , , , , , | Leave a comment

Until the Twelfth of September

Posted on September 12, 2022 by melelllan

Well, what a day it is. Five days earlier than expected, ACADIA announced that the FDA has accepted the application for Trofinetide! There’s a lot to go over in this announcement, so let’s cut to the chase! “SAN DIEGO–(BUSINESS WIRE)–Sep. … Continue reading →

Posted in Acadia, FDA, hope, International Rett syndrome Foundation, LAVENDER TRIAL, margaret brimble, Neuren Pharmaceuticals, Rett Syndrome, RSRT, Trail to a Texas Trial, Trofinetide, Trofinetide Phase III, Uncategorized | Tagged , | 5 Comments

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

ASCEND 2022!

Posted on May 11, 2022 by melelllan

After a VERY long hiatus, the annual/biannual Rett syndrome Family Conference came back with a BANG! The backdrop being the incredible, but entirely too large, Gaylord Resort, Nashville. Thank you to IRSF for putting together such a massive and comprehensive … Continue reading →

Posted in Acadia, Anavex, Ascend Rett syndrome conference 2022, boys with Rett syndrome, clinicaltrials.gov, Communication, David Clements, Dr. Helen Leonard, Dr. Walter Kaufmann, FDA, fund-raising for rett syndrome, hope, Huda Zoghbi, Kevin Black, LAVENDER TRIAL, Raising a Hand, Rett Research, rettsyndrome.org, Telethon for Kids, Trail to a Texas Trial, Trofinetide, Trofinetide Phase III, Uncategorized | Tagged , , , , , , , , , , , , , , , , , | Leave a comment

A Chat with James Shaw of Neuren

Posted on January 6, 2022 by melelllan

Talking with James Shaw, Vice President, Clinical & Regulatory Operations, is like talking with an old friend. He has been there every step of the way through Katelin’s journey with Trofinetide and his dear heart is so invested in our children. The few times … Continue reading →

Posted in Acadia, Dame Margaret Brimble, Intrepid 2566, LAVENDER TRIAL, Neuren Pharmaceuticals, Rett Syndrome, rettsyndrome.org, TBI, Trail to a Texas Trial, Trofinetide, Trofinetide Phase III | Tagged , , , , , | 4 Comments

A Chat with Dr. Pichard of IRSF

Posted on December 20, 2021 by melelllan

Disclaimer: Unless specifically stated, the possible benefits down the line are my opinion alone. As one might imagine, I and other parents have a ton of questions since the results of the Trofinetide Phase III came out. One of the … Continue reading →

Posted in Acadia, anxiety in Rett syndrome, Breathing issues in Rett syndrome, clinicaltrials.gov, Communication, Daffodil trial, Dame Margaret Brimble, Dr. Helen Leonard, LAVENDER TRIAL, Neuren Pharmaceuticals, Rett Research, Rett Syndrome, rettsyndrome.org, screaming in rett syndrome, Talking, Trofinetide Phase III | Tagged , | Leave a comment

It’s a wrap!

Posted on November 12, 2021 by melelllan

Neuren announces the completion of the Phase III Trofinetide study (Lavender). We are now on a countdown to results. You can read about it- here I want to thank everyone who participated in any way, for any amount of time … Continue reading →

Posted in Acadia, LAVENDER TRIAL, Neuren, Trail to a Texas Trial, Trofinetide | Leave a comment

The Home Stretch!

Posted on August 4, 2021 by melelllan

The Pandemic has interfered with so many aspects of our lives, in general; for me, it has taken away all my time to write as Katelin has been out of her day program for nearly 16 months. That ended last … Continue reading →

Posted in Acadia, LAVENDER TRIAL, Neuren Pharmaceuticals, Trail to a Texas Trial, Trofinetide, Trofinetide Phase III | Tagged , , , , , , | 2 Comments

Sites are Within Sight!

Posted on April 1, 2021 by melelllan

It may have been easy, during the last year, to lose sight of an end to the Trofinetide trial. Nothing about this last year has really been easy, except losing hope. I want to thank all the families that have … Continue reading →

Posted in Acadia, clinicaltrials.gov, LAVENDER TRIAL, Neuren Pharmaceuticals, Rett Research, Rett Syndrome, Trail to a Texas Trial, Trofinetide, Trofinetide Phase III | Leave a comment