Tag Archives: Rett syndrome clinical trials

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

Patient Focus Drug Development Meeting

Posted on March 19, 2022 by melelllan

On March 11, the global Rett Community had an extraordinary opportunity- to educate the FDA about the severe consequences of having a child with Rett syndrome- for the child, the family, the community and society. As we listened to the … Continue reading →

Posted in boys with Rett syndrome, FDA, Rett syndrome Awareness | Tagged , , , , , , , , , , , , | 2 Comments

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

Trofinetide Series-Part 2: LAVENDER-Study Description and Study Design

Posted on January 20, 2020 by melelllan

Alrighty then! Time to start breaking down the information on ClinicalTrial.gov. It’s going to take a while to get through all the info, however I will try to keep in concise and understandable. The key, here, is that the more … Continue reading →

Posted in Acadia, clinicaltrials.gov, FDA, Neuren Pharmaceuticals, Orpan Drug Status, Rett Syndrome, Trail to a Texas Trial, Trofinetide, Trofinetide Phase III | Tagged , , , , , , | 2 Comments

Rett Syndrome is Such a Trial… #2 Ketamine

Posted on May 15, 2019 by melelllan

Many parents can attest to the fact that after a procedure requiring anesthesia our children have a decrease in Rett related symptoms. For Katelin, this was a drastic decrease in behavioral issues. My other children would often say… Can we … Continue reading →

Posted in Breathing issues in Rett syndrome, FDA, ketamine, Rett Research, Rett Syndrome, Rettland Foundation, rettland.org, RSRT, Trail to a Texas Trial, Uncategorized | Tagged , , , , , , , , , | 2 Comments

Rett Syndrome is Such a Trial… #1 Anavex 2-73

Posted on April 12, 2019 by melelllan

…and 2019 looks to change that up a bit- Rett Syndrome, so many trials! According to the most recent GP2C newsletter (GirlPower2Cure)there will be at least 7 trials this year, currently in various stages. This is SO exciting! What does … Continue reading →

Posted in Anavex, Anavex 2-73, cure, Dr. Walter Kaufmann, FDA, gp2c, hope, Orpan Drug Status, Rett Research, Rett Syndrome, Rett syndrome conference 2016, Rettland Foundation, rettland.org, rettsyndrome.org, Trail to a Texas Trial, Uncategorized | Tagged , , , , , , , , , , | 3 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 trofinetide 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

All is Not Quiet on the Trofinetide Front #1 A Chess Game

Posted on January 6, 2019 by melelllan

It’s been very quiet in the public eye in regard to Trofinetide. We all are in a holding pattern as we wait for Phase III to begin, but BEHIND the scenes things are progressing strategically and clearly, like a chess … Continue reading →

Posted in Acadia, anxiety in Rett syndrome, Ataxia, boys with Rett syndrome, breakthrough therapy, Communication, cure, FDA, Fragile X, hope, margaret brimble, Neuren Pharmaceuticals, Rett Research, Rett Syndrome, rettland.org, rettsyndrome.org, Trail to a Texas Trial, Trofinetide, Trofinetide Pediatric trial, Trofinetide Phase III, Trofinitide, Uncategorized | Tagged , , , , , , , , , , , , , , , , , , , , , | Leave a comment

Winding Down

Posted on October 30, 2018 by melelllan

Rett syndrome Awareness #30 October is winding down and we’ve almost made it through the month. As I sit here and write this, I know there are families who didn’t make it through, they lost their children in the days … Continue reading →

Posted in boys with Rett syndrome, cure, fund-raising for rett syndrome, Huda Zoghbi, margaret brimble, Neuren Pharmaceuticals, Rett Research, Rett Syndrome, Rett syndrome Awareness, rettland.org, rettsyndrome.org, RSRT, Trail to a Texas Trial, Uncategorized | Tagged , , , , , , , , , , , | Leave a comment