Download January, 2009 Dear Friends, Rett Syndrome strikes without warning

January, 2009
Dear Friends,
Rett Syndrome strikes without warning. The girl with Rett syndrome starts out as a happy, healthy baby until
12-18 months of life. Then something goes terribly wrong. She loses the ability to speak. She can’t use her
hands to play with toys, feed herself or wave hello. She may lose the ability to walk, she makes constant hand
washing or hand wringing movements. By the time she is 3 years old, the girl with Rett syndrome is severely
handicapped. Rett syndrome is the only autism spectrum disorder with a known genetic cause. The genetic
mutation that causes Rett syndrome was found in 1999 and in 2007 researchers reversed the symptoms of Rett
syndrome in mice models! Research is urgently needed to bring about treatments and a cure for this
devastating disorder.
Dr. Anthony Horton joins International Rett Syndrome Foundation (IRSF) from the Alzheimer’s Drug Discovery
Foundation as our new Chief Scientific Officer. He is leading a comprehensive research initiative to establish a
3-5 year scientific roadmap outlining milestones leading to treatments and a possible cure of Rett syndrome.
For the year, IRSF’s research commitment exceeds $1.7 million and IRSF’s cumulative total support of research
projects is now over $18 million dollars! According to Dr. Horton, “Today marks the beginning of an aggressive
new strategy that will lead research forward in the search for treatments and a cure for Rett syndrome. In
addition to these high-quality proposals - submitted by some of the most prestigious labs in the field - we will
now begin to broaden our research portfolio to focus on several large scale projects in early stage drug
discovery with a view to catalyze the development of sub-type specific therapeutics targeting different RTT
mutations.”
Currently, another part of this important research, is the ongoing NIH funded Natural History Study of girls and
women with Rett Syndrome. Here in New Jersey, we are in our third year of fostering this important initiative.
We have been very fortunate to host some of the world’s leading Rett syndrome specialists here at UMDNJ
Robert Wood Johnson Medical School in New Brunswick, NJ. Families are able to consult with Rett specialists
from two of our nation’s most renowned Rett syndrome centers at Baylor University in Houston, TX and the
University of Alabama in Birmingham, AL, without having to travel. Presently, over 100 families from NJ as well
as NY, CT, PA, & MD are enrolled in this exciting program.
Due to the huge response to this study clinic, and the families’ responses to our needs survey, we are working
with UMDNJ Robert Wood Johnson Medical School to create a permanent Rett Syndrome Clinic program here.
By operating a Rett clinic we can provide an effective health care system that will encourage effective
diagnosis, treatment and clinical care to our children in one central location, so their functioning can be
maximized and additional problems prevented.
The 17th annual Silent Angels Gala to benefit Rett Syndrome will be held on March 28, 2009 at the Twin Brooks
Country Club in Watchung NJ. Your contribution will allow us to help fund this important research and make
this clinic a reality. Please review the enclosed contract for ways you can help. We now have hope, that there
will be a cure for Rett syndrome, and we thank you for helping us move closer to our goal.
Thank you in advance for your generosity.
Very truly yours,
Leslie Greenfield
Executive Director