Wow, these last two weeks have been an absolute whirlwind when it comes to Rett syndrome research. To start with, Neurogene announced on January 23, 2023 that they had received approval from the Food and Drug Administration (FDA) in the United States to begin human clinical trials of their gene therapy drug, NGN-401. This was an absolute shock to the community considering that it's been less than a year since Neurogene announced plans to begin developing a drug.
Neurogene Initial Announcement (May 18, 2022) - https://www.neurogene.com/press-releases/neurogene-announces-new-development-program-in-rett-syndrome-utilizing-novel-exact-technology-platform/
Neurogene EXACT Technology Explained - https://www.neurogene.com/our-technology/
Rett Syndrome Research Trust sits down with Neurogene - https://youtu.be/QuTu_zLcVoI
We also continue our conversation with Dionne Snyders by discussing more specifics of what it's like running a school with such a diverse student population. To learn more about Nova School, visit their website.
https://www.nova-school.co.za/
If you are a newly diagnosed family, you can find loads of information regarding Rett Syndrome on our website (linked below), including links to various foundations and support groups worldwide. And, of course, feel free to reach out to us; we love connecting with new families!
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