Category Archives: Orpan Drug Status

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

The Next Steps Through the Desert

Wow. While time has been passing, Neuren has quietly, behind the scenes,  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

It’s Official!

This morning Neuren released a statement in regards to Orphan Drug Status for Trofinetide (NNZ 2566) See their announcement here. They’ve also come out with an awesome video about the science behind Trofinetide. Take a look! Great graphics and pretty … Continue reading

Posted in FDA, Neuren Pharmaceuticals, NNZ-2566, Orpan Drug Status, Trofinetide | Tagged , , , , , , | Leave a comment