Category Archives: Trofinetide

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

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

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

A Crystal of Happiness and Hope

There is a poem I love. It serves to remind me that we are all entitled to the “Pursuit of Happiness” but also that you must learn to be content with any pieces you find because, in truth, no one … Continue reading

Posted in hope, Poetry, Rett Syndrome, Rett syndrome Awareness, Trail to a Texas Trial, Trofinetide, Trofinetide Phase III, Uncategorized | Tagged , | 2 Comments

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

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

Trofinetide Series-Part I: Words you Need to Know

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 Challenge Accepted! ;)

It hasn’t happened before. I was given a MISSION, if I chose to accept it, to explain some new developments in regards to Sarizotan (Newron) and Anavex 2-73 (Anavex) receiving FDA Rare Pediatric Disease Designation: “Mel Lancaster have you posted … Continue reading

Posted in Acadia, Anavex, Anavex 2-73, FDA, Neuren, Neuren Pharmaceuticals, Newron, Orpan Drug Status, sarizotan, Trail to a Texas Trial, Trofinetide | Tagged , , , , , , , | Leave a comment

A Day with ACADIA- Part 2: Assurance

Katelin and I had the great pleasure of being invited to ACADIA a couple of weeks ago for Rett syndrome Awareness Month. Last week I posted Part 1: Awareness; I had PLANNED to follow that closely with Part 2, but … Continue reading

Posted in Acadia, Dame Margaret Brimble, Education, Neuren Pharmaceuticals, Rett Research, Rett Syndrome, Rett syndrome Awareness, Trail to a Texas Trial, Trofinetide, Trofinetide Phase III | Leave a comment