Rett Syndrome has advantages over other disease groups. Rett has a known genetic cause, in most cases, mutations in a single gene, MECP2, and reversibility has already been demonstrated in a mouse model of the disease.
Reversibility - not prevention, but reversibility, reversal of symptoms even in mice of late stage disease.
Since MECP2 was identified as the cause of Rett in 1999, research has made great strides in understanding the underlying cause of Rett Syndrome, finding ways to treat symptoms and figuring out how to translate the reversal experiments into a human therapy, with the help of some of the world's best scientists.
Reverse Rett works collaboratively with partners worldwide, to compound efforts to drive this research forward, ultimately accelerating the delivery of treatments and a cure for Rett Syndrome and related MECP2 disorders.
We fund international Rett Syndrome research through our longstanding partnership with the US based, Rett Syndrome Research Trust (RSRT) who are responsible for peer review and monitoring of those projects.
Our UK work has a translational impact with clear patient benefits. We work to ensure that as treatments and a cure for Rett Syndrome become viable, they are accessible to patients in the UK. To date, the focus of our UK work has been supporting the research and clinical objectives of a single site at King's College London, where the first UK clinical trial for Rett Syndrome started earlier this year.