Category Archives: Rett Research

Rett syndrome Independence Day Gets (hopefully) a Little Closer! Trofinetide Reopens- An Interview with Dr. Jim Youakim

On Saturday June 27, I had the pleasure of speaking with Dr. Jim Youakim, Vice President of Research and Development, to get an update on the trofinetide trial and it’s recent reopen. Bill Keller, Executive Director of Patient Advocacy, joined … Continue reading

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

ACADIA Talks Trofinetide and Covid-19: An Interview with Dr. Jim Youakim

Recently, I was given the incredible opportunity to interview Dr. Jim Youakim, Vice President of Research and Development. This was a bit out of my wheelhouse, but he and Bill Keller, Executive Director of Patient Advocacy, were very gracious in … Continue reading

Posted in Acadia, clinicaltrials.gov, hope, Neuren Pharmaceuticals, Rett Research, Rett Syndrome, Rett syndrome Awareness, rettsyndrome.org, Trail to a Texas Trial, Trofinetide Phase III | Tagged , , , , , | Leave a comment

An Update on Trofinetide Coming Soon to a Blog Near You!

This past weekend, I had the great honor of being chosen to interview  Dr. Jim Youakim. Dr. Youakim heads up the Trofinetide trial from Princeton. I thought I’d give you a preview of his qualifications and some of his thoughts … Continue reading

Posted in Acadia, Neuren Pharmaceuticals, Rett Research, Rett Syndrome, rettsyndrome.org, Trail to a Texas Trial | Tagged , , , , , | 1 Comment

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

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 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

Close is Too Late for Too Many

As we approach the start of the Trofinetide trial, I know that it is going to go by in a blink.  And, there are other trials in different stages of development, each holding some parent’s hope for their child- improvement … Continue reading

Posted in Acadia, Anavex, Avexis, boys with Rett syndrome, cure, Death in Rett syndrome, grief, Neuren Pharmaceuticals, Rett Research, Rett Syndrome, Rett syndrome Awareness, tracy chapman, Trail to a Texas Trial, Trofinetide Phase III | Leave a comment