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Category Archives: FDA
Daybue, A Year in Review
I’m sorry I’m 3 days late. I should have had this post to GO! But blog writer fail there. March 10, 2023, the day that ended the “no treatment” era of Rett syndrome. It’s largely true that the first couple … Continue reading
The Day the Flowers Bloomed in Oz
Not many know this, but I am a children’s author (ok, it’s only two books, but it counts!) and I’m very proud of that. I am also a published poet and my greatest honor was being asked to write a … Continue reading
Posted in Acadia, Dame Margaret Brimble, FDA, hope, Neuren, Rett Syndrome, Trail to a Texas Trial
Tagged Children's story, Larry Glass, Neuren stockholders
1 Comment
Daybue’s Debut Conference Call by Acadia
Disclaimer *Any comments on data are mine alone and are not confirmed nor authorized by Acadia* Acadia held an investor’s conference call today on the FDA approval of Daybue (trofinetide). While the call was for investors, there were several areas … Continue reading
Posted in Acadia, Acadia Connect, boys with Rett syndrome, Daybue, FDA
Tagged Kathie Bishop
1 Comment
BLAST OFF!!!! Part 1
It would be hard to believe that anyone in the Rett community has not heard the news- Daybue (trofinetide) was approved by the FDA yesterday!!! This is TREMENDOUS news for all of us. You can read Acadia’s official announcement- HERE … Continue reading
Posted in Acadia, Acadia Connect, Daybue, FDA, Trofinetide, Uncategorized
1 Comment
T – 14 (or less) Musing
By all counts the FDA will announce at most 14 days from today. March 12 being a Sunday it can go either way- the Friday before or the Monday after. Of course, they can announce any day now. In my mind, I’ve likened the last 6 months to a pregnancy. How it just seems to drag and drag at the beginning. You do your best to prepare, but what if you don’t know if it’s a boy or a girl? or if it’s twins? You know, like before the days of ultrasound ;). So much unknown, but you do have a due date- March 12! Continue reading
Posted in Acadia, Dame Margaret Brimble, FDA, hope, Neuren Pharmaceuticals, Rett Syndrome, Trofinetide
Tagged dreams, Hubble, Milestones, Webb
2 Comments
T-20 Musing: Parents’ Helping Hands
I often see parents who comment on social media that they feel disappointed, or defeated, etc. because they were unable to participate in a clinical trial. But, it takes all types of participation for a trial and ultimate approval (hopefully!!) … Continue reading
Time to Wrap it Up
Today is a date that might pass by without much thought for most, not me. Someone told me today they “live for deadlines”. Well, today’s a special deadline. With March 12 being the PDUFA date (the date the FDA has … Continue reading
Posted in Acadia, FDA, Rett Syndrome, Trail to a Texas Trial, Trofinetide
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Timelines, Lifelines, and Lifetimes
Today marks three months into the FDA process of the review of Trofinetide, the halfway mark (of course it could always be sooner!). Taking a look at the timeline for NDA priority review, it shows that at this point the … Continue reading
Posted in Acadia, Death in Rett syndrome, FDA, grief, Poetry, Trail to a Texas Trial, Trofinetide
Tagged Ted Moreland
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One Month Down, Five to Go!
Time is blowing in the wind. Did you feel it? Did it seem so long? Was it an awful wait, those 30 days? It was nothing but a whisper that flew past. It was a speeding wish making its way … Continue reading
Until the Twelfth of September
Well, what a day it is. Five days earlier than expected, ACADIA announced that the FDA has accepted the application for Trofinetide! There’s a lot to go over in this announcement, so let’s cut to the chase! “SAN DIEGO–(BUSINESS WIRE)–Sep. … Continue reading
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