Monthly Archives: February 2015

Rare

Posted on February 28, 2015 by melelllan

Originally posted on Colleen English:
Today is Rare Disease Day and this year’s theme is: Day-by-day, hand-in-hand. I personally love that there is a strong movement of many coming together, from all corners, representing thousands of different diseases, all raising…

Posted in Uncategorized | Leave a comment

Rare Disease Day

Posted on February 28, 2015 by melelllan

Today, Feb. 28, is Rare Disease Day. Around the world, “Day by day, hand in hand” people face these rare diseases and hope for cures. There are about 7000 known rare diseases in the world. In the US a disease … Continue reading →

Posted in Rett Syndrome, Trail to a Texas Trial | Tagged , , , , , | Leave a comment

BTD-1…..GO!…..Musing 3

Posted on February 27, 2015 by melelllan

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

Posted on February 26, 2015 by melelllan

I am a researching junkie. True. Difficult to believe, I know. 😉 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

BTD-3 (Breakthrough Therapy Designation Countdown) Musing 1-Ready

Posted on February 25, 2015 by melelllan

One of the new things Katelin is saying is “Ready, Set….GO!” Ready? Oh, yes I am. I’m ready for my daughter to have a chance. I’m ready to jump for joy with all the other Rett parents who are ready … Continue reading →

Posted in Uncategorized | Leave a comment

Please, Please, Please, Please

Posted on February 24, 2015 by melelllan

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

Posted on February 19, 2015 by melelllan

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 →

Posted in Army of us, FDA, Rett Syndrome, Trail to a Texas Trial | Tagged , , , | Leave a comment

February, the new October

Posted on February 18, 2015 by melelllan

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!

Posted on February 16, 2015 by melelllan

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

Army of Us Challenge

Posted on February 14, 2015 by melelllan

As many of you know Duncan Millar and James Westgate are two of my all time favorite people I’ve never met in real life. They are phenomenal. So they put out a call to load a picture and list three … Continue reading →

Posted in Army of us, rett uk | Tagged , | 2 Comments