New Research Trust Dedicated to Furthering Treatment of Rett Syndrome Launched in the United Kingdom

A new non-profit organisation, the Rett Syndrome Research Trust UK (RSRT UK) launches today. Set up by the families of girls and women with Rett Syndrome, the new trust has a sole focus – namely to fund research and capitalise on recent scientific advances in order to ensure Rett Syndrome becomes the first ever reversible brain disorder. RSRT UK will work closely with a US partner, the Rett Syndrome Research Trust.

Rett Syndrome is an autism spectrum disorder which manifests in apparently healthy little girls, just as they are beginning to speak and walk. Rett Syndrome robs them of those emerging skills, stagnating their development and leaving them with a myriad of disabilities and complex medical needs for the rest of their lives. There is no cure.

But researchers know that Rett Syndrome is caused by mutations in a gene called MECP2. This gene makes a protein which is required for proper maintenance of neuronal function. Most astoundingly, recent research has demonstrated that if proper levels of the protein are restored, the symptoms of Rett Syndrome are reversed in mouse models of even late-stage disease.

The organisation’s inaugural event, a gala reception is planned for November 18, 2010. The event will be held at the Wellcome Trust Headquarters in London, with guest of honour, Professor Adrian Bird of the University of Edinburgh, the world’s leading expert in MECP2, Trustee and scientific advisor to the Rett Syndrome Research Trust and author of the landmark paper in the journal Science, which established the reversibility of Rett Syndrome in mice.

Rachael Bloom, Chairman of the Board of Trustees of RSRT UK and mother of a 14-year-old daughter who suffers with the disorder, said: “The Rett Syndrome Research Trust UK formed when a group of UK families came together with the fervent belief that parents must take an active role in the fight against Rett Syndrome. We want to see this research driven to its ultimate conclusion, replicating the results of the reversal experiments not in mice, but in girls and women living with Rett Syndrome today.

Time is not on our side. A cure that is decades away may come too late for families of adult girls, and condemns little girls, newly diagnosed, to a childhood burdened by the staggering array of neurological and orthopaedic symptoms which arise as Rett Syndrome progresses. Our partnership with the US-based Rett Syndrome Research Trust, our shared name and common goal, puts us in a prime position to aggressively impact research and the speed at which treatment can be delivered for all of our daughters, no matter where in the world they may be.”

Commenting on the launch of RSRT UK, Executive Director of the US-based RSRT Monica Coenraads said: “This syndrome devastates the lives of children all over the world. The determined commitment of a global network of families is crucial if the promise of reversal is to be achieved in our lifetimes. We applaud the dedication, energy and foresight of the parents spearheading RSRT UK and look forward to working with like-minded families everywhere who share our urgency, focus and determination to reverse Rett.”