The Rett Syndrome Research Trust, a newly established nonprofit organization, is pleased to announce the launch of our operations and website (www.rsrt.org). In the wake of last year's dramatic pre-clinical reversal of Rett symptoms, the Trust has been formed to pursue the next steps toward treatment and cures for Rett Syndrome and the increasing range of MECP2 spectrum disorders. Our short-term goal is to bring novel therapeutics addressing the underlying MECP2 pathology to clinical trials within five years.
Rett Syndrome is the most physically disabling of the autism spectrum disorders and the leading cause of severe genetic impairment in females, with an estimated 30,000 individuals afflicted. At this time the number of children born with other MECP2 spectrum disorders remains unknown. In the U.S., the frequency of autism has risen to 1 in 150 children.
Classic Rett Syndrome affects females almost exclusively. Symptoms typically begin to appear in little girls between infancy and two years of age. Onset may be heralded by hypotonia and delayed development, or by regression and loss of acquired skills, including speech and functional hand use. As the disorder progresses, symptoms may include disordered breathing, sleep disturbances, severe digestive problems, orthopedic abnormalities, anxiety, apraxia, seizures, impaired cardiac and circulatory function and Parkinsonian tremor. Many children with Rett Syndrome are wheelchair bound. Most victims survive well into adulthood; all require total, 24-hour-a-day care. The cost, both financial and in terms of human suffering, is enormous.
The vision of the Rett Syndrome Research Trust is guided by founders and advisors who have been responsible for many of the major advances in Rett research over the past decade. Based on their comprehensive analysis of strategic priorities at our inaugural international meeting, RSRT's initial projects build the foundation for a drug discovery platform
|Contact: Monica Coenraads|
Rett Syndrome Research Trust