Category Archives: NNZ-2566

Epic Fail (Not)

Today was an epic fail on my part. And so I will generalize this day and trials, not just Trofinetide, but just in general. I started out with a GREAT premise…Katie would LOVE a whale watch. We were on one … Continue reading

Posted in cure, Intrepid 2566, Neuren, Neuren Pharmaceuticals, NNZ-2566, TBI, Trail to a Texas Trial, Trofinetide, Uncategorized | Leave a comment

Three more Colors of Dawn

As Rett syndrome Awareness Month comes to a close, I want to bring to attention two other disorders… Fragile X and Traumatic Brain Injury. What do they have to do with Rett syndrome? … Trofinetide and a chance. In the … Continue reading

Posted in cure, FDA, Fragile X, fund-raising for rett syndrome, hope, Intrepid 2566, Neuren Pharmaceuticals, NNZ-2566, Rett Syndrome, TBI, Trofinetide, Uncategorized | Tagged , , , , , , , , | 1 Comment

Newsworthy News!

Today, like many other Rett parents, I got an e-mail from Neuren in regards to their Chairman’s Address to Stockholders. It was chock full of hopeful expectations. Of particular note: “Finally, I would like to make some remarks in relation … Continue reading

Posted in Ann Romney Center for Neurological Diseases, Brigham and Women's, cure, Neuren Pharmaceuticals, NNZ-2566, Rett Research, Rett Syndrome, rettland.org, TBI, Trofinetide | Tagged , , , , , , , , , , | 1 Comment

A Call to Arms!

We have yet another chance to influence the course of Rett syndrome research! Paige Nues, Director of Family Empowerment, sent out an email…In part it says: As the result of meetings just held in Washington DC, many of our elected … Continue reading

Posted in Congress, Intrepid 2566, NNZ-2566, Peer Reviewed Medical Research Program, rettsyndrome.org, TBI | Tagged , , , , , , , , , , , , , , , , , | 2 Comments

BTD-1…..GO!…..Musing 3

Today, as we reach the 60 day limit from application to hopefully an announcement. I have a simple post. Our wishes have been READY…. . . . . . . . . We’ve been SET for a long time…. . … Continue reading

Posted in breakthrough therapy, FDA, Neuren, NNZ-2566, Rett Syndrome, rettsyndrome.org, Trail to a Texas Trial, Trofinetide | Tagged , , , , , , , | Leave a comment

February, the new October

As most who read this blog know, October is Rett Syndrome Awareness month. But this month has seen its share of awareness, too. I was honored to give a presentation on Rett Syndrome and our NNZ trial experience to three … Continue reading

Posted in NNZ-2566, 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