Monthly Archives: February 2019

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,, RSRT, Trail to a Texas Trial, Trofinetide, Uncategorized | Leave a comment

My Wish to the Stars

Our little community has lost at least 5 girls and one mother to Rett syndrome and FoxG1 since the start of the new year. Facebook is a double edged sword-you watch these children grow, rejoice in new skills or coming … Continue reading

Posted in Death in Rett syndrome, FoxG1, Poetry, Rett Syndrome, Trail to a Texas Trial, Uncategorized | Leave a comment

To the Doctor that Pissed Me Off

I read this article today titled, To the Nurse Who Made Me Cry and it asks at the end, “Fellow Mighty friends, if you could write a letter to a medical professional, what would you say?” Now, I’ve actually often … Continue reading

Posted in acceptance, Communication, Education, grief, hand clasping in Rett syndrome, hope, Rett Syndrome, Rett syndrome Awareness, screaming in rett syndrome, Trail to a Texas Trial, Uncategorized | Leave a comment

The Next Steps Up the Mountain

Neuren Pharmaceuticals and Acadia have announced more details about the upcoming Phase III trial. Read about them HERE. Two very important blurbs stick out: “The Phase 3 study will be followed by an open label extension study in which all … Continue reading

Posted in Acadia, FDA, Neuren Pharmaceuticals, Rett Research, Rett Syndrome, Rett syndrome Awareness,,, Trail to a Texas Trial, Trofinetide, Trofinetide Phase III | Leave a comment