Ivan J Garel-Jones via Crowdrise
January 19, 2011
BENEFITING: Rett Syndrome Research Trust
EVENT DATE: Mar 20, 2011
I am running the LA Marathon on March 20th 2011 in support of the Rett Syndrome Research Trust.
What is Rett Syndrome?
Rett Syndrome is the most physically disabling of the autism spectrum disorders. It strikes at random in early childhood, affecting little girls almost exclusively. Many girls live into adulthood, requiring total, 24-hour-a-day care. There is no treatment beyond supportive, and often ineffective, measures such as feeding tubes, bracing, orthopedic and GI surgeries, and medications for anxiety and seizures. First recognized only 25 years ago, the prevalence of Rett Syndrome equals that of Cystic Fibrosis, Huntingtons and ALS but is vastly underfunded in comparison to those disorders
Why am I supporting this Charity?
I know a family that has a daughter with Rett Syndrome. I have two daughters. I wanted to help.
Why should you support it?
See below. Scientists were able to reverse the effects of the disease in mice in 2007. If a cure can be found for Rett's it could also have knock on effects for other forms of autism and schizophrenia.
What does the Research Trust do?
The goal of the Rett Syndrome Research Trust is to deliver clinical trials of a novel treatment strategy within five years. The Rett Syndrome Research Trust ia 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.
What if I want to read more?
Here are some links