Category Archives: Orpan Drug Status

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

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

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

Sarizotan Trial #2 A Bit of Background

So, I was going to talk about how Sarizotan is supposed to work to help with daytime apnea this week, but I thought I’d give a bit of background on it instead. šŸ™‚ Back in 2002-2003, Sarizotan was used in … Continue reading

Posted in anxiety in Rett syndrome, Breathing issues in Rett syndrome, Newron, Orpan Drug Status, orphan drug designation, Rett Research, Rett Syndrome, rettland.org, sarizotan, screaming in rett syndrome, Trail to a Texas Trial, Uncategorized | Tagged , , , , , , , , , , , | Leave a comment

Trofinetide #3

And on we go! So far I’ve discussed what Trofinetide is, how it came about and what it does. Great, but what are we talking for a timeline, right? Who holds the strings? Just what the heck are the steps … Continue reading

Posted in boys with Rett syndrome, breakthrough therapy, FDA, hope, margaret brimble, Neuren Pharmaceuticals, NNZ-2566, Orpan Drug Status, orphan drug designation, Rett Syndrome, rettland.org, Trail to a Texas Trial, Trofinetide, Trofinetide Pediatric trial, Trofinetide Phase III, Uncategorized | Tagged , , , , , , , | 20 Comments

The Next Steps Through the Desert

Wow. While time has been passing, Neuren has quietly, behind the scenes, Ā been taking step after step and gaining ground with Trofinetide. Check out their latest announcements… http://www.neurenpharma.com/IRM/ShowDownloadDoc.aspx?SiteId=386&AnnounceGuid=23a3caf0-969f-4269-824e-fe32548eefa9 http://www.neurenpharma.com/IRM/ShowDownloadDoc.aspx?SiteId=386&AnnounceGuid=89e7ec87-31e5-4aa3-b029-0b449df3189c So, to me it feels like we went from full stop … Continue reading

Posted in Congress, cure, Neuren Pharmaceuticals, Orpan Drug Status, Rett Research, Rett Syndrome, rettsyndrome.org, Trail to a Texas Trial, Trofinetide | Tagged , , , , , , | Leave a comment