US THEN 2016
The words we have been waiting for…
FDA approves Trofinetide for treatment of RETT SYNDROME! This is big news! This isn’t a cure but it is a treatment. Will it work for everyone with Rett Syndrome? No. Will it work for Magnolia? We aren’t sure. Are we happy about it? We are thrilled! Its a huge accomplishment. The amount of work that Rett families , Rett specialists and RettSyndrome.org had to do to get here will not be forgotten.
This trial has been a pivotal force in our lives. We made a documentary about our journey. We changed life perspective from our experience.
Magnolia was in Phase 2 of the clinical trial in 2016. We didn’t see improvements then. We assumed she was on the placebo because we were hearing whispers that some families were seeing improvements. It was a hard but exciting time. The trial was emotionally grueling for us. It was hard to continue when we didn’t see any improvements. It is hard to continue putting your trial through a clinical trial when you realize they might be the child getting pricked with needles for seemingly no reason. Halfway through we knew we had to continue. I really will never be able to put into words the toll it took. The problem is, for drugs to get approved, clinical trials have to be completed. We would never get to FDA approval for treatments or gene therapy if we can’t’ finish trials.
Phase 3 of the trial started 3 years later! We wanted to put Magnolia in it. If there is an opportunity to give her something that helps her, even just 10%. We need it for her. The problem was in 2019 Magnolia was having severe GI issues, was diagnosed failure to thrive, and having severe seizures. Her body couldn’t take much more. To do the trial, you have to be fairly stable. She wasn’t. We were in and out of hospitals. It was a scary time.
But here we are in 2023 and Trofinitide has been approved by the FDA. It will help some. We will try it again.
I am so happy and proud of the families that persevered. Each phase of the trial meant something, but there is no denying the stress of the last phase. Phase 3, which started right before the pandemic in 2020 and has lasted throughout the past 3 years, definitely must have been the most difficult to navigate.. Those families deserve a hug from every single one of us. THANK YOU!
For all future reference TROFINITIDE will now be called DAYBUE. We are anticipating the debut of DAYBUE.