In 1999, RSRT Scientific Advisory Board member Huda Zoghbi established the link between the MECP2 gene and Rett Syndrome. In 2007, RSRT Trustee, Adrian Bird, dramatically reversed Rett Syndrome symptoms in mice.

Launched in the wake of this unexpected result, RSRT has a single focus — to make Rett Syndrome the first reversible brain disorder. The speed of our success will, in large part, be dictated by the magnitude of our financial investments in high quality research. The scientists, technologies and ideas are in place — the next steps are clearly defined. Our children cannot wait decades for a cure.

RSRT is a 501c3 nonprofit research organization exclusively 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 our founders and advisors, some of the world’s most brilliant scientific minds, who are largely responsible for the major advancements in Rett Syndrome research. The Trust is currently supporting a portfolio of key research projects including the first foray into drug development for Rett Syndrome. The Trust has also launched a series of international scientific think tanks.

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 debilitating syndrome includes symptoms seen in many other severe neurological and neuropsychiatric disorders on which Rett research may shed light:

• Regression, including loss of speech, motor control and functional hand use
• Autonomic instability and sleep disturbances
• Autistic behaviors and sensory issues
• Impaired cardiac, circulatory and digestive functions
• Parkinsonian tremors
• Many varieties of seizures, often untreatable
• Anxiety
• Apraxia
• Dystonia
• Orthopedic problems including scoliosis and osteopenia

Currently there are no effective treatments for Rett Syndrome. Most girls survive into adulthood and require total, 24-hour-a-day care.

Mutations in a gene called MECP2 cause Rett Syndrome and have also been found in individuals diagnosed with autism, learning disabilities, anxiety disorders, childhood onset schizophrenia, and other neuropsychiatric disorders. Research on Rett Syndrome will have far-reaching implications for a myriad of disorders.

In 2007 the journal Science published the work of Adrian Bird, Ph.D. demonstrating the reversal of Rett Syndrome in mature mouse models with late stage disease. Only days away from death, these animals recovered normal function and became indistinguishable from healthy mice in a matter of weeks.

This singular achievement has catapulted Rett into new realms of possibility and positions Rett Syndrome to be the first curable childhood neurological disorder.

The future of children and adults with Rett Syndrome depends on research. Without it, they will be completely dependent and locked in for the rest of their lives. The Rett Syndrome Research Trust is supported by Rett Syndrome families and friends who agree with the urgency of our cause and wish to see the science move forward full speed ahead.

The field of Rett research has exploded since Prof Bird’s breakthrough work. We now need an army of informed and committed supporters to help us see the damage of Rett Syndrome reversed not in mice, but in humans.

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.