Tag Archives: Neuren Pharmaceuticals

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 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 Mother Meets a Child She Will Give New Life To

It is no secret that I have high expectations for Trofinetide, I fully expect Katelin will speak; I fully expect her to be able to read and write; I expect that she will be calm, well she is Irish, so … Continue reading

Posted in Acadia, ACS Medicinal Chemistry Division, American Chemical Society, Dame Margaret Brimble, hope, Mark Brimble, Neuren Pharmaceuticals, NNZ-2566, NNZ-2566 trial, Rett Syndrome, TBI, Trail to a Texas Trial, Trofinetide, Trofinetide Pediatric trial, Trofinetide Phase III | Tagged , , , , , , , , , , | 7 Comments

Lavender and Lilac

Allow me to introduce you to the code names of the Trofinetide Phase III! Makes me think of the time I was in the VP’s motorcade and we all had code names, lol. First up we have Lavender- a 12 … Continue reading

Posted in Acadia, cure, Neuren Pharmaceuticals, Rett Research, Rett Syndrome, Trail to a Texas Trial, Trofinetide, Trofinetide Phase III | Tagged , , , , , | 1 Comment

All is Not Quiet on the Trofinetide Front #2; A Balancing Act

* 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

A Little Trip, A LOT of Hope!

A few weeks ago, Katelin and I were invited to go to Acadia! On a whim, I googled “Acadia” to see what might popup. First up, beautiful Acadia National Park. Definitely would like to go there! Actually, I can’t believe … Continue reading

Posted in Acadia, cure, FDA, fund-raising for rett syndrome, hope, Neuren, Neuren Pharmaceuticals, Rett Research, Rett Syndrome, Rett syndrome Awareness, rettland.org, Trail to a Texas Trial, Trofinetide, Trofinetide Pediatric trial, Trofinetide Phase III, Uncategorized | Tagged , , , , , , , , , , , , | 3 Comments