Study update – Week 3

If you want to know how Week 1 and Week 2 went, check out those links.

I wish I had something amazing to say about our study experience but so far…we are just not seeing any major changes here. As much as I tried to prepare myself for the possibility of getting the placebo, I was really, really, really hoping for something else. As much as I am reminding myself that we very possibly might be on the placebo a new fear is creeping in…

What if he’s getting the full dose, and it doesn’t work for him?

Of all the possibilities that I ran through in my head, I knew that this was one of them. But knowing it and knowing it are very different once you’ve left the land of “what if” and are standing smack dab in the middle of “what is.”

Not all of the boys on the study have improved on this drug. We knew that from the stories people had shared and we knew it from the researchers and FX experts we’ve seen. This drug is doing amazing things for some of the kids participating on the trial but no one ever, ever, ever said this would be the cure…that this would fix it.

As a parent, I want nothing more than for someone “in the know” to give me some guarantees but they can’t. In all likelihood, this will help some but not all. In all likelihood, we’re still looking at a combination of drugs to fix what our one little broken gene has done. It’s supremely frustrating.

I keep reminding myself to be patient, that we are less than 2 months away from knowing for sure what the outcome will be on this particular drug. That whispering voice is becoming more and more distracting though. I find myself gazing out the window lost in thought trying to remember what it was like before. I find myself watching Monkey’s every move trying to decide if he’s doing more or less of that particular behavior. It’s enough to make a Mama crazy, and this Mama really didn’t need any help in that department!

51 more days. We’ve got this.

15 thoughts on “Study update – Week 3

  • November 14, 2012 at 11:32 am
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    Oh, honey. I think I wrote this very same blog post a year ago. We didn’t notice a lot of changes until around the final 3 weeks of the study – at which point the boys were weaned from whatever they were on – the drug, or the placebo, or a combination of both. What we noticed was that some of the negative behaviors we hadn’t even really noticed had disappeared, came back. We’ve been on the extension for 10 months now, and we realize the changes are subtle, but they are there. I wish someone had told me to settle down and stop looking so hard. I know that’s next to impossible to do. You’re right – it’s not going to miraculously, overnight, fix that broken gene. But it may do some good repairs, given time. In the meantime you are taking part in a great thing – you are promoting research and study to improve the lives of all our kids!

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  • November 15, 2012 at 2:23 pm
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    We are in the same boat. Should be heading for the “taper down” now, but really never saw any change. I appreciate the post from Bonnie about seeing the subtle changes later – maybe that will be the case here too. It’s eas to tell yourself you’re not going to get your hopes up…it’s much harder to realize that you had them high to begin with!

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