Rett Syndrome Research Trust, Inc.

Imagine the symptoms of autism, cerebral palsy, Parkinson's, epilepsy, and anxiety disorders....all in one little girl

Now imagine these symptoms vanishing...one by one

This is the work of the Rett Syndrome Research Trust

We welcome  your commitment and support

 

A cure is possible
In 2007, University of Edinburgh Professor Adrian Bird demonstrated that mice terminally ill due to MECP2-deficiency become healthy again if the gene is restored.  This remarkable discovery proved that the Rett-like phenotype can be reversed even in late stages of the disease.  There is now proof that Rett is a curable condition.  But a significant investment in Rett research is needed to turn this promising result into treatments and a cure for girls and women with Rett. 

Rett Syndrome Research Trust—leading the charge to find a cure
The Rett Syndrome Research Trust (RSRT) encourages and supports the most promising scientific research on Rett Syndrome.  To carry out its goal of finding a cure for Rett, RSRT has recruited some of the most prominent neuroscientists and geneticists to the Rett Syndrome cause.  RSRT has been instrumental in funding, at least in part, most of the seminal Rett research projects to date.  Thus far, an investment of $20 million has yielded a number of exciting potential treatment approaches.  RSRT’S lean structure allows 97% of all support to be channelled directly to research.

What is Rett Syndrome?
Rett Syndrome is the most physically disabling of the autism spectrum disorders. Primarily affecting little girls, Rett often strikes just after they have learned to walk and say a few words, and begins to drag their development backward. This debiliating syndrome includes symptoms seen in many other severe neurological disorders on which Rett research may shed light. Currently there are no effective treatments for Rett. Most girls survive into adulthood and require total, 24-hour-a-day-care.

Links to other disorders
Mutations in the "Rett gene" have also been found in individuals diagnosed with autism, learning disabilities, anxiety disorders, childhood onset schizophrenia, and other neuropsychiatric disorders.  Research on Rett will have far-reaching implications for a myriad of disorders.

MECP2 Duplication/Triplication Syndrome
Unlike Rett Syndrome which is caused by mutations or deletion in MECP2, the duplication or triplication syndrome is caused, as the name suggests, by having an area of the X chromosome which includes MECP2, erroneously duplicated or tripled. Families of these children together with RSRT have created the MECP2 Duplication Syndrome Fund at RSRT.  100% of all fund raised supports research on the duplication/triplication syndromes.