Being a part of history

Looking back, it seems so long ago that Maggie was a part of the Trofinitide clinical trial. At 5 years old, my little girl went through 10 weeks of weekly appointments in a different city, to change her life. How many people can say that? I wish she didn’t have to do it, I wish so many things were different for her, except for one. Her bravery. She is brave. Two things…She is also strong. Just like so many other girls with Rett Syndrome, she chooses to fight to change her life.

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I’m not sure we have discussed this, because life is a blur these days. We talk to Maggie about everything. We explain everything to her, about Rett, about trials, about life. During the trial, there were a couple of moments, we almost quit. It was really hard on us, and really hard on her. There were times she wanted to quit, mostly during the bloodwork, but she prevailed. She would tell us she didn’t like the Doctor, but loved the nurse. She hated the guy that took her blood, but liked the other nurse. She was very specific about who she liked working with. There were times, I almost picked her up and walked out of the clinic. In the end, I’m glad that we did it. We didn’t find out if she was on the drug or on the placebo. We are fairly positive she was on the placebo, because we really didn’t see any change in her. Actually, things got worse for her during that time period. To go through a trial on a placebo (possibly) but still have to endure the hardships of the clinical trial really tested us. We just kept telling ourselves and her, someone has to go through the trials, actually we hope everyone participates. If everyone quit when they didn’t see results, we’d get nowhere. And we want Rett to be history. So we endure.

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The good news is, Neuren Pharmaceutical released an announcement today. From the way we are reading the results, it looks like the drug works. What?!  The next Phase of the trial is set for 2018, which feels so very far away, but with everything that she has been through lately, I’ll take the good news. It also looks like the drug helps symptoms across the board. Every step forward with Rett treatments and cures, I’ll celebrate.

For most people, its hard to understand why being a part of a clinical trial is hard. That’s fine, I hope you never have to fully understand, just take my word for it. The travel, the mental exhaustion, worry about the drug and its effects and not to mention the monetary aspect. Thanks to Rettland and their mission to help families participate in clinical trials. Also, luckily, Maggie’s uncle Sean was able to get a house for us, for most of the weekends, and we were able to stay at the same place for most of the trial, which Maggie loved. Thank you Uncle Sean and Aunt Lynn. Maybe Uncle Sean and Aunt Lynn will buy her that house, because she really really loves it there.

Looking back at the clinical trial, as hard as it was to travel and upend our lives almost every weekend for two and a half months. I’m so glad that we were able to find balance. Maybe it’s San Diego, but our down time away from the clinic, was pretty awesome.

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To read Neuren’s press release regarding Trofinitide, go here.Press Release

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