Category Archives: rettsyndrome.org

Acadia Prepares for the Triple Crown

It’s been a long haul for all of us waiting on this final phase of Trofinetide trials. It’s like waiting through the Triple Crown… First, the Kentucky Derby! Such a big deal, it’s crazy the hoopla that surrounds it. Then, … Continue reading

Posted in Acadia, Neuren Pharmaceuticals, Rett Research, Rett Syndrome, Rettland Foundation, rettland.org, rettsyndrome.org, Trail to a Texas Trial, Trofinetide, Trofinetide Pediatric trial, Trofinetide Phase III | Tagged , , , , | 2 Comments

Rett Syndrome is Such a Trial… #1 Anavex 2-73

…and 2019 looks to change that up a bit- Rett Syndrome, so many trials! According to the most recent GP2C newsletter (GirlPower2Cure)there will be at least 7 trials this year, currently in various stages. This is SO exciting! What does … Continue reading

Posted in Anavex, Anavex 2-73, cure, Dr. Walter Kaufmann, FDA, gp2c, hope, Orpan Drug Status, Rett Research, Rett Syndrome, Rett syndrome conference 2016, Rettland Foundation, rettland.org, rettsyndrome.org, Trail to a Texas Trial, Uncategorized | Tagged , , , , , , , , , , | 3 Comments

Hold the Presses-Trofinetide in the News!

The first publication of the Pediatric Trofinetide trial has hit the proverbial newsstand! Neurology.org has published “Double-blind, randomized, placebo-controlled study of troļ¬netide in pediatric Rett syndrome” ahead of print. So, it’s hotter than “hot off the press.” So, quick cut … Continue reading

Posted in Acadia, anxiety in Rett syndrome, Ataxia, Breathing issues in Rett syndrome, Communication, cure, Dr. Walter Kaufmann, hand clasping in Rett syndrome, Intrepid 2566, margaret brimble, Neuren Pharmaceuticals, Neurology.org, NNZ-2566, Orpan Drug Status, Rett Research, Rett Syndrome, Rett syndrome Awareness, rettsyndrome.org, screaming in rett syndrome, seizures in Rett syndrome, Texas Children's Hospital, Trail to a Texas Trial, Trofinetide, Trofinetide Pediatric trial, Uncategorized | Tagged , , , , , , , , , , , , , , , , , , , , | Leave a comment

International Women’s Day

I’ve had some great female role models over the years and I have a pretty long memory. The first woman I remember, even before my own grandparents, was Grandma Watson, we lived upstairs from her when I was just a … Continue reading

Posted in chan zuckerberg initiative, huda zoghbi, Huda Zoghbi, International Women's Day, margaret brimble, Rett Research, Rett Syndrome, Rett syndrome Awareness, rettland.org, rettsyndrome.org, RSRT, Trail to a Texas Trial, Uncategorized | Tagged , , , , , , , , , , , , , , | Leave a comment

I Love Someone Rare

Today is Rare Disease Day 2019 From the NIH website: What is a rare disease? In the United States, a rare disease is defined as a condition that affects fewer than 200,000 people. This definition was created by Congress in … Continue reading

Posted in Acadia, Anavex, Anavex 2-73, Avexis, FDA, Neuren Pharmaceuticals, Orpan Drug Status, orphan drug designation, Rett Research, Rett Syndrome, rettsyndrome.org, RSRT, Trail to a Texas Trial, Trofinetide, Uncategorized | Leave a comment

The Next Steps Up the Mountain

Neuren Pharmaceuticals and Acadia have announced more details about the upcoming Phase III trial. Read about them HERE. Two very important blurbs stick out: “The Phase 3 study will be followed by an open label extension study in which all … Continue reading

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

Up To The Mountain-again.

I struggled to come up with a title for this post and I struggled in deciding whether to write it or not. But, I challenged myself to be honest with both myself and others when I began this blog and … Continue reading

Posted in Acadia, anxiety in Rett syndrome, Communication, cure, Neuren Pharmaceuticals, Phone, Rett Research, Rett Syndrome, rettland.org, rettsyndrome.org, Talking, Trail to a Texas Trial, Trofinetide, Trofinetide Phase III | 6 Comments