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Daybue, A Year in Review
Posted on March 14, 2024 by melelllan
I’m sorry I’m 3 days late. I should have had this post to GO! But blog writer fail there.


My FB profile picture March 10, 2023
March 10, 2023, the day that ended the “no treatment” era of Rett syndrome. It’s largely true that the first couple of years or so after a new drug comes out is the fourth phase; having a treatment, when it’s the ONLY treatment, means it’s not THE treatment for everyone. This has become sadly apparent. People have struggled with the main side effects- diarrhea, vomiting, and weight loss.

Daybue has been hailed in the circles where stockholders and pharmaceutical companies and medical journals reside; it has been cautiously advanced by doctors who were thrown by having to deal with diarrhea vs constipation for the first time; it has been decided against by doctors who know their patients can’t take a chance at losing a pound or are not mobile enough that aspiration pneumonia is a real concern. It has been defamed by biased, innuendo laced pseudo-reports. And so, what do we really have?

We have the first treatment for Rett syndrome. Like all medications, not everyone can tolerate it. We have a community of ingenious people who are finding all sorts of ways to make it so their children can take it without or, at least limited, side effects. Moreover, we KNEW this would be the case. More than doctors, more than Acadia (AcadiaConnect and AnovoRX have really stepped up to this challenge), parents and caregivers are the line in the sand; that line of defense that has to work it out- and many, many have.

But let’s start with Acadia– they went all out with AcadiaConnect and after a very short, rocky start, gave us all an asset through our FAM’s (Family Access Managers) who are our contacts for concerns and questions. A shout out to my FAM, Ruben, who checks in every week and remains my sounding board. They gave us AnovoRx, which is available 24/7 (8-8 and after hours on call pharmacist). AnovoRx has done a stand-up job of listening to what worked and didn’t work for diarrhea, coming up with new suggestions as doctors and caregivers provided feedback. This really is a great service.

Acadia continues its mission of educating new parents through its ongoing “Discovering DAYBUE™ (trofinetide) Caregiver Series”-the next one is coming up on March 21, 2023. Reach out to AcadiaConnect or your FAM with questions before or after. I attend each of these and am happy to answer any questions as well.


And the biggest news of all, Acadia was given RoW (Rest of the World) rights to Daybue AND NNZ-2951 for Rett syndrome and Fragile X. NNZ-2951 is considered by many to be the golden ticket of molecules as it is being tested by Neuren in four other rare diseases.

You can see where each drug is in the process. Dame Margaret Brimble created both these molecules. Neuren and Acadia stand poised to bring the treatments of six rare diseases to fruition, tremendous really.


Taken directly from Neuren’s website. All rights reserved by them.
It was a mad scramble at first, in every way-from the rollout, to trying to manage the side effects, to seeing incredible results or, in some cases, no changes. For my part, I have run the gamut of confused, distraught, determined, frustrated, and profoundly surprised many times. But after a year, things are calming down. The major issues are known; interventions have changed for the better, Acadia is listening to caregivers and working on their end to address our concerns.

And in the words of Katelin, spoken yesterday to the bus driver, for the very first time- you have a nice day!

For those hoping for a year in review of Katelin’s Daybue journey, she didn’t start until June, so it’s coming



https://trailtoatexastrial.wordpress.com/2024/03/14/daybue-a-year-in-review/