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Rett Syndrome – RSRT


Rett Syndrome is a devastating neurological disorder that affects primarily girls and women. More than 350,000 are afflicted around the world. Children with Rett develop normally in the first year of life. Then, tragically, they regress, with most losing the ability to speak and walk. A host of other painful and debilitating symptoms ensue. The cause of Rett is known—a random mutation on a single gene—setting it apart from other neurological disorders and giving researchers a target. Remarkably, Rett has been reversed in mouse models, and research is now focused on translating that achievement to girls and women with the disease. With continued research progress and sufficient funding, Rett Syndrome could become the first neurological disorder to be cured.


A non-profit called the Rett Syndrome Research Trust (RSRT) is leading the charge to develop a cure for Rett. Since it was founded in 2008, RSRT has awarded over $47 million to leading scientists to advance Rett research to the exciting stage it is now in. RSRT is a lean and efficient non-profit. An average of 96% of funding is spent directly on research. In 2017 RSRT announced a bold, three-year, $33 million strategic research plan called Roadmap to a Cure. The plan prioritizes four scientific approaches that are most likely to lead to a cure. Gene therapy, the lead program of Roadmap to a Cure, is making particularly promising progress. Informed by data generated by RSRT-supported researchers, a biotech company called AveXis is now developing the first human clinical trial in gene therapy for Rett Syndrome.


RSRT’s funding needs to carry out this bold research are significant. All of RSRT’s support comes from individual donors who want their dollars to have an impact.

To contribute, go to or send a check made out to “RSRT” to:


67 Under Cliff Road, Trumbull, CT 06611


RSRT is a 501c3 non-profit. Donations are tax deductible. For more information please contact Tim Freeman at tim(at)rsrt(dotted)org or 1.609.309.5676.