Category Archives: orphan drug designation

Even Still, We Must Reach for the STARS

Hyperventilation, daytime apnea, breath-holding– whatever name you call it by, it’s a horrific breathing pattern that severely impacts the quality of life of many of our children and their families. In 2016, Newron Pharmaceuticals undertook the STARS trial, an international … Continue reading

Posted in clinicaltrials.gov, Newron, orphan drug designation, rettsyndrome.org, sarizotan, 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

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. 🙂 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