Category Archives: nnz 2566

In the Spotlight!- Dame Margaret Brimble

Posted on March 26, 2019 by melelllan

Soon the Phase III Pivotal trial for Trofinetide will begin. I have a personal goal of full recruitment within three months. That doesn’t mean everyone has started, but that all the spots are ready to be filled. WE CAN MAKE … Continue reading →

Posted in cure, Dame Margaret Brimble, hope, Neuren Pharmaceuticals, nnz 2566, NNZ-2566 trial, Rett Research, Rett Syndrome, Rett syndrome Awareness, Trail to a Texas Trial, Trofinetide, Trofinetide Phase III, Uncategorized | Tagged , , , , , , , , , , , , , , , , , , , | Leave a comment

20 Years in Oz

Posted on October 1, 2015 by melelllan

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 , , , , | 4 Comments

And….the President Answers

Posted on March 30, 2015 by melelllan

  Dear Melinda: Thank you for writing, and for your service and sacrifice. I have heard from too many Americans whose lives have been affected by a wide range of health conditions and diseases. Whether they are common, preventable ailments … Continue reading →

Posted in nnz 2566, Rett Syndrome, Trail to a Texas Trial, Trofinetide, white house | Tagged , , , | Leave a comment

The Pen is Mightier than the Denial

Posted on March 3, 2015 by melelllan

Soooo…… I haven’t reached the “Let it Be” part of my last post, just yet. I tried, I took lots and lots of deep breaths, before my neuromuscular disease kicked in and I needed to stop that. Now, I’m doing … Continue reading →

Posted in Neuren, Neuren Pharmaceuticals, nnz 2566, Rett Syndrome, Trail to a Texas Trial, Trofinetide | Leave a comment

One Step Closer

Posted on February 14, 2015 by melelllan

It is just after midnight and my girl is sleeping (thank goodness!!) and I wonder if her and other Rett children’s dreams just became filled with butterflies. Why butterflies? Because they are to me a most beautiful symbol of metamorphosis, of … Continue reading →

Posted in FDA, Neuren, nnz 2566, Rett Syndrome, Trofinetide | Tagged , , , , , , , , | 4 Comments

Tick Tock, Hurry up Clock!

Posted on February 9, 2015 by melelllan

Katie’s favorite author, alas, is not me; it’s Dr. Seuss. So, in the spirit of Dr. Seuss…. Tick Tock, hurry up clock. There is a place known as the Land of Rett and in the middle of town, there’s a … Continue reading →

Posted in nnz 2566, Rett Syndrome, Talking, Trail to a Texas Trial, Trofinetide | Tagged , , , , , , , | 4 Comments

The Road from Here to There- the next 28 steps

Posted on February 1, 2015 by melelllan

Well, we are counting down now, 28 days or less, 28 steps or less, ’til we turn another bend in a treatment for Rett syndrome; the FDA’s time to respond to Neuren’s application for Orphan Drug Status and Breakthrough Therapy … Continue reading →

Posted in FDA, Neuren, nnz 2566, Rett Syndrome, Trail to a Texas Trial | Tagged , , , , , , , , | Leave a comment

What’s in a Name?

Posted on January 28, 2015 by melelllan

In a lifetime, names are some of the most important words we’ll ever know. Picture that expectant mom and dad- sometimes they’ve known for years what they wanted to name their baby-a family name, a favorite relative, a junior, a … Continue reading →

Posted in Neuren, nnz 2566, Rett Syndrome, Trail to a Texas Trial, Trofinetide | Tagged , , , , | 7 Comments

Soon, Just a Phone Call Away

Posted on January 25, 2015 by melelllan

How simple a thing a phone call seems. Katie’s sister sends her videos on the phone for her to watch, and Katelin listens when her sister calls, but not once have they had a conversation, except when Katie was one … Continue reading →

Posted in Communication, Neuren, nnz 2566, Phone, Rett Syndrome, Talking, Trail to a Texas Trial | Tagged , , , , , , | Leave a comment

One Person can Change the World- Be the Light

Posted on January 24, 2015 by melelllan

Martin Luther King Day got me to thinking about how one person can change the world; they really can. He did. So did plenty of other people, all in their own way, some  great, some small. Some I don’t know … Continue reading →

Posted in Neuren, nnz 2566, Rett Syndrome, Trail to a Texas Trial | Tagged , , , , , , , , , | 1 Comment