We are Nearing the True Unknown


Boldly going into the unknown

So, we are closing in on the third and final pivotal trial of Trofinetide. It is my understanding that Neuren and the FDA are meeting in October. Sometime next year the study will begin recruiting and start. What the criteria are, the end goals are etc. are still not published as far as I know.

But, because there is definitely a lot of buzz about it, parents are beginning to really contemplate-What will Trofinetide mean for my child?!?

I think that’s a very natural question and fear, actually. I believe I know to some extent what it means for Katelin, but at over 5x the dose she received (or didn’t receive) I can only guess. I mean 5x the dose! So, I think- will she do 5x more in the same short time? or does it cap out for older girls somewhere? I had a glimpse and so did Katie. I know she wants a phone and a car, a black one. I think you can guess which she’ll get, lol. But, her articulating to me that she believes she will talk and wants a phone is HUGE. So, is the fact that she wants a car, but even if Trofinetide fixed EVERYTHING she’s only 4.5 ft tall, I don’t think she can reach the pedal, thank goodness. But, parents are wondering other really significant things.

My child never talked-what kind of therapy is  going to be needed.

Will it be like starting over with a baby?

My girl is 40 or older, do I give it to her?

There’s so much unknown, there’s so much Hope, and there’s fear.

Fear that it won’t work on a particular mutation; that it won’t work on their child; that it WILL work.

These are all understandable fears. Even I, a true believer in what may be possible, am a bit afraid.

Afraid? you say. You! Oh, yes.

I Am afraid of the unknown-how will Katie’s and my relationship change if she can talk? What if she says she doesn’t want to live at home? She has been my focus for 23 years, controlled to some extent everything I do and when I do it. What if she can’t come to terms with the fact that she’s had to use diapers/pull-ups as an adult and realizes not everyone does that. How will she feel when she gets angry and doesn’t resort to banging her head, only to realize the damage she’s done over the years. How will she feel about herself in this (almost certain) new awareness of herself, her disability, her memories. How will she feel when she looks at the scars on my arms and knows that she put them there? How will she develop a new relationship with her siblings-one not based on disability but equality of language and memory. Will she forgive them for their resentment, will they accept her and forge a new relationship with her. One thing I know for sure is that if Katelin can talk, we are going to a counselor. She will need help to process what is happening to her, to us as a unit, and any possible past trauma she can finally speak about.

But, what I do NOT fear is showing her the world and taking her lead about what she may want to learn, where she may like to go. I’ve been in “Holland” so long, I’m excited about finally maybe going to Italy or Ireland or the next state over without worrying. I look forward to being able to go somewhere alone, knowing that she can tell me everything that happens while I’m gone. No more scared to death of what the untold might hold.

There is always a lot to fear in the unknown. It’s like those first astronauts stepping into a teeny tiny capsule that would take them to the moon and back. Really, that “back” must’ve been in their minds all the while, no matter how much faith they had in the science. How brave they were. How brave WE are, our children and us, to allow ourselves to face such a rare opportunity- to truly dare.

So, as we approach this deadline that we have waited and waited for, bravery is called for. We must be brave enough to face our fears. Maybe that means not giving Trofinetide to a woman who’s 40 or 50 when all their lives their parents have hoped for the day. Maybe it means facing that “empty nest” you never thought you would have. In the end, we don’t know what any of us may be facing. All the trials have been too short really, we just won’t know until we get there. Much like being new parents-you never know what life holds in store for you and your new child, but oh the promise that can be.


This entry was posted in acceptance, boys with Rett syndrome, Communication, cure, FDA, hope, Neuren Pharmaceuticals, Rett Research, Rett Syndrome, Talking, Trail to a Texas Trial, Trofinetide, Uncategorized and tagged , , , , , , . Bookmark the permalink.

3 Responses to We are Nearing the True Unknown

  1. Rose and Renée says:

    Great insight, thoughts about things I never considered. Happy thoughts!!?

  2. Linda E. Williams says:

    Hi to my long-distance fiend. Missing you. So much hope for oh so long… May your hopes be realized for a new life, a new set of dreams, once never expected, be set in motion, and peaceful nights of sleep become routine. God bless and keep you well and busy Love and hugs, L

  3. Linda E. Williams says:

    May you and Katie have a warm, safe
    Christmas and an amazing new year filled with hope and achievements long prayed for. Love and prayers go out to you. L

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