Tag Archives: Neuren Pharmaceuticals

T-27/30

Posted on February 12, 2023 by melelllan

Depending upon whether the answer comes on the 10th or the 13th, this is where we are; T-30 at worst (barring any unforeseen circumstances). And so, I begin my musings. What does it feel like, for me, to be 30 … Continue reading →

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Neuren Talks Trofinetide and Rest of the World

Posted on September 28, 2022 by melelllan

For those of us in the States, we’ve got a pretty clear picture of the next few months, the FDA will spend the next 5.5 months reviewing the data and will give ACADIA and us an answer on or around … Continue reading →

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17 Hopes of September

Posted on September 4, 2022 by melelllan

Our children suffer, greatly, and parents/caregivers are right beside them on that journey. When your child is born you have so many great hopes for them, dreams beyond compare. Then, Rett syndrome, the thief in the night, steals that all … Continue reading →

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17th of March in September

Posted on September 2, 2022 by melelllan

Katelin was in the NNZ-2566 (Trofinetide) trial through the holidays the year she was in Texas. We were incredibly blessed. One family was having a party for Thanksgiving at the hotel we were in and shared their meal with us. … Continue reading →

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Trofinitide: A Parental Case Study

Posted on June 21, 2022 by melelllan

As the trail to Trofinitide is coming to its conclusion- the long awaited will they/won’t they approve, I want to revisit Katelin’s experience during the trial, when parents weren’t sworn to silence. It’s been a long, nearly nine years and … Continue reading →

Posted in Acadia, Neuren Pharmaceuticals, NNZ-2566, NNZ-2566 trial, Trail to a Texas Trial, Trofinetide | Tagged , , , , , , , | 3 Comments

Rett Syndrome Awareness Month #1 Horizons

Posted on October 2, 2021 by melelllan

Life is made up of journeys, reaching for horizons, goals that appear almost out of reach-that final mountain you can barely see the tip of, way on out over there, all hazy and colorful. We all start out pretty much … Continue reading →

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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

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

Posted on May 7, 2020 by melelllan

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!

Posted on April 13, 2020 by melelllan

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

Posted on January 17, 2020 by melelllan

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