About

Welcome to the Rett Syndrome Research Trust. We are a 501(c)(3) nonprofit organization intensively focused on the development of treatments and cures for Rett Syndrome and related MECP2 disorders. The strength of the Trust is based on the guidance of founders and advisors who are largely responsible for the major advances in Rett research over the past decade.

In February of 2007, the journal Science published a paper by Adrian Bird, Ph.D., demonstrating the reversal of Rett Syndrome in mature mouse models with late stage disease.  This remarkable and unexpected result has established a new paradigm for Rett Syndrome and other neuropsychiatric conditions, such as autism and schizophrenia.

The obvious and urgent question the Trust seeks to answer is whether symptoms can be reversed, and normal function restored, in people suffering with Rett Syndrome and MECP2 spectrum disorders.  To this end, the Trust has convened a series of international think tanks and developed a comprehensive, strategic portfolio of projects, including the first foray into drug discovery for Rett Syndrome. This plan was developed in concert with, and will be executed by, distinguished scientists who are thought-leaders in their fields.

Neither the government, through the National Institutes of Health (NIH), nor pharmaceutical companies can be relied upon to finance the work that lies ahead. NIH funding mechanisms are not designed to quickly support highly innovative exploration. Pharmaceutical companies will only engage once early drug discovery and clinical development have been undertaken.

A private effort is essential if treatments and cures are to be developed in a time frame that the devastating nature of this disorder demands. RSRT is catalyzing such an effort and invites you to join our cause.