Category Archives: FDA

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

20 Years in Oz

I saw a writing contest advertised on Facebook, the very next ad under it was something to do with the Wizard of Oz…and so I was inspired. I had hoped to get this published, but no takers. However since this … Continue reading

Posted in cure, FDA, nnz 2566, Rett Syndrome, Trail to a Texas Trial, Trofinetide, Uncategorized, Wizard of Oz | Tagged , , , , | 3 Comments

Tonight should be the Day

If the FDA is on target with their 60 days, Neuren should have been notified over the weekend; so, around 6 pm or 7 pm US EST, we should know what the FDA has said about Breakthrough Therapy Designation. Over the … Continue reading

Posted in FDA, Neuren Pharmaceuticals, Rett Syndrome | Tagged , , , , , | 5 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

BTD-2……..Set…… Musing 2

I am a researching junkie. True. Difficult to believe, I know. đŸ˜‰ I like being “set”. I like knowing what I’m getting in for, whether it’s figuring out my daughter had Rett syndrome before any doctor would say “Your daughter … Continue reading

Posted in breakthrough therapy, cure, FDA, Neuren Pharmaceuticals, Rett Syndrome, rettsyndrome.org, Trail to a Texas Trial | Tagged , , , | 2 Comments

Please, Please, Please, Please

One “please” for evey day left. Rett parents say so many “pleases” that never get answered. Please, don’t let that MecP2 test come out positive. Please, don’t let my baby lose her words. Please, let me hear my baby say … Continue reading

Posted in breakthrough therapy, FDA, hope, Neuren Pharmaceuticals, Rett Syndrome | Tagged , , , , | 2 Comments

TO(GET)HER STRONGER

Monday was President’s Day in America, honoring George Washington and Abraham Lincoln. Of course, they deserve to be honored-one for forging a Nation and the other for saving it. Another mom was asking for inspirational quotes and it brought to … Continue reading

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