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Right Place
We are the organization working to cure Rett Syndrome. As the largest worldwide funder of Rett research, we're advancing genetic medicines that target the root cause of Rett. The gene therapy programs currently in clinical trials were started and incubated at RSRT. We are the go-to partner for genetic medicine scientists and companies.
The commitment and passion of Rett families in the US and around the globe make our critical work possible. If you want a cure as urgently as we do, we welcome you to our site and invite you to join us in our quest.
The Next Wave of Genetic Medicines for Rett
Our June webinar drew a record number of participants for a look at where the science stands right now: the biotechs entering the field, the delivery breakthroughs making treatment possible, and when the next clinical trials for Rett could begin.
Monica Coenraads and the RSRT research team walked through every active program and closed with an in-depth Q&A. The full recording is now available.
Our goal is to put ourselves out of business as quickly as possible.
Rare diseases don’t cure themselves. Developing genetic medicines is a complex, expensive effort with many moving parts. We are the organization orchestrating this global effort. Here is our plan to cure Rett syndrome.
Recently Diagnosed?
Getting a Rett syndrome diagnosis is one of the hardest things you’ll ever face. We know this first-hand. Emotions can run the gamut and it can all seem overwhelming. You’re worried about your child and your family. You’re reading everything you can get your hands on but unsure how to interpret it or if it's relevant. We’ve been there, just reach out to us: we’ve got your back.
The Summit
Over the course of three days in September we convened leading scientists, clinicians, and regulators to explore every corner of the genetic medicines landscape. The leadership from over 45 biopharma companies had a unique opportunity to hear about the state of Rett genetic medicine programs, with the ultimate goal of inspiring and motivating them to add the disorder to their pipelines.
This year’s gathering underscored RSRT’s role as both convener and catalyst, bringing brilliant minds together while never losing sight of the children and families at the heart of our community.
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MECP2 Duplication
MECP2 Duplication syndrome (MDS) is caused by a genetic error that duplicates a section of the X chromosome that includes the MECP2 gene. The MECP2 Duplication Syndrome Fund at RSRT, created in 2010, puts the intellectual and scientific resources of RSRT immediately to work for the MDS community.
