Big Deal

Today some big news came out and it’s a pretty big deal for our family. You can click here to read for yourself, it is a press release that announces Orphan Drug status from the FDA for trofinetide as a treatment for Rett syndrome. This news brings with it a lot of questions. What does it do? How will it help? To most people who don’t have a degree in neurobiology it’s a little overwhelming.

It’s hard to understand why it is a big deal because nobody is talking about how great it is. The thing is, fewer than 50 families were in the clinical trial. The families that were in phase 2 of the trial were blinded. Nobody knew who was getting the drug or the placebo and to protect the study, details of what happened while in the study aren’t to be discussed.

It’s hard to get excited about something that you’ve never seen work. It’s easier for me. I’ve seen it work. I don’t know for sure that Claire was on the drug last year while she completed the first half of the IGF-1 trial but whatever was in those injections was good. I can’t tell you how good or what changes we saw. I don’t think it matters.

Better is better.

The time I’ve spent in Rettland has worn me down and I am no longer picky. I once had specific hopes for Claire. Now I will take anything that helps at all. That is what this news is. Something that will help. Today’s announcement means that we will be able to learn more about how it can help sooner than later. We have to wait on the regulatory process for everyone’s safety, but help is on it’s way.

From our walk today, making the most of what we have, anxiously looking forward to the day that we get just a little more Claire.
From our walk today, making the most of what we have, anxiously looking forward to the day that we get just a little more Claire.

5 thoughts on “Big Deal

  1. Colleen, this makes my tiny little arm hairs quiver with delight and excitement. I am so proud of you for your contributions to making this happen. You are a pioneer of awesome!

  2. Cathi says:

    Well done! I love reading your blog it makes me feel less alone! Thank you for helping this to come about I can’t wait. Xx Cathi ( mother to Saffron 7 who has Rett Syndrome)

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