Category Archives: Neuren Pharmaceuticals

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

Hot off the Presses- The Horses are Outta the Gate!

Well yesterday’s post WAS going to be the last one this month but then I got an email from Acadia- “Hot off the Presses” it said and then this announcement- ACADIA Pharmaceuticals Initiates Phase 3 Pivotal Study of Trofinetide in … Continue reading

Posted in Acadia, Dame Margaret Brimble, hand clasping in Rett syndrome, margaret brimble, Neuren, Neuren Pharmaceuticals, Rett Research, Rett Syndrome, Rett syndrome Awareness, rettsyndrome.org, Trail to a Texas Trial, Trofinetide Phase III, Uncategorized | Leave a comment

A Day with Acadia- Part 1: Awareness

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 Trip of a Lifetime

I recently took a trip with Katelin. A long, boring, exhausting trip- more than worth it for the end result, but the getting there and getting back were tough. Katelin threw up on the way back and that was the … Continue reading

Posted in Acadia, Communication, cure, hope, Neuren Pharmaceuticals, Rett Research, Rett Syndrome, Rett syndrome Awareness, rettsyndrome.org, screaming in rett syndrome, Special needs siblings, Talking, Trail to a Texas Trial, Trofinetide, Trofinetide Phase III | 2 Comments