Rett Syndrome (RTT) is a debilitating neurological disorder diagnosed almost exclusively in females. Children with RTT appear to develop normally until 6 to 18 months of age when they enter a period of regression, losing speech and motor skills. Most develop repetitive hand movements, irregular breathing patterns, seizures and extreme motor control problems. RTT leaves its victims profoundly disabled, requiring maximum assistance with every aspect of daily living. There is no cure... yet.
Every 5 hours somewhere in the world, a child is born with Rett syndrome. Rett syndrome affects 1 in 10,000-15,000 live female births and there are over 200,000 girls and women worldwide who have Rett syndrome. Rett Syndrome knows no geographic, ethnic or socio-economic boundaries. Any expectant parent is at risk of having a child with Rett syndrome.
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The IRSF Strollathon is the signature event of the International Rett Syndrome Foundation*. It is a fun-filled event which will help us raise crucial research dollars and also increase public awareness of RTT here in the Roanoke Valley. Ours is one of more than 25 Strollathons to be held this year across the country! Last year's Grand Total from 21 Strollathons across the country was an incredible 1.1 Million Dollars!
*IRSF is the world's leading private funder of basic, translational and clinical Rett syndrome research, funding over $28M in high-quality, peer-reviewed research grants and programs to date. Annually, IRSF hosts the world's largest gathering of global Rett researchers and clinicians to establish research direction and priorities while exchanging ideas and the most recent information. IRSF is the most comprehensive non-profit organization dedicated to providing thorough and accurate information about Rett syndrome, offering informational and emotional family support and stimulating research aimed at accelerating treatments and a cure for Rett syndrome and related disorders.IRSF has earned Charity Navigator's most prestigious 4-star rating.
SAVE THE DATE:
The 9th Annual
Strollathon for Rett Syndrome
Saturday, September 14, 2013
at Wasena Park in Roanoke.
Registration starts at 10:00
Stroll begins promptly at 11:00
Countdown to Strollathon 2013
Rett syndrome is the only autism-spectrum disorder with a known genetic cause,
Board of Directors of the International Rett Syndrome Foundation have approved
funding in the amount of $1.3M to support new grants for translational research
ranging from clinical studies and developing outcome measures to preclinical
studies of new compounds in mouse models of Rett syndrome (RTT). Two ANGEL
grants were awarded in this first cycle of the Translational Grant Program this
- The first ANGEL award will fund Phase I and Phase IIa of the NNZ-2566 clinical study in adult patients with Rett syndrome. This study will be spearheaded by Dan Glaze, MD and Jeffrey Neul, MD, PhD in conjunction with Neuren Pharmaceuticals. With IRSF funding, Baylor College of Medicine and Neuren are well-positioned to move forward with this clinical study upon FDA approval of the IND.
- The second ANGEL award to N. Carolyn Schanen, MD PhD, Nemours Biomedical Research and Jeffrey Neul, MD PhD, Baylor College of Medicine and the Jan and Dan Duncan Neurological Research Institute entitled Nonsense suppression as a therapeutic approach to Rett syndrome will fund a pre-clinical study of two compounds that may suppress MECP2 mutations that result in short forms of the MeCP2 protein. These types of mutations are found in 30-35% of all those with MECP2 mutations. Therefore, these compounds have the potential to help a great number in the RTT population.
Three HeART grants were also awarded in this
first cycle of the Translational Grant Program. These grants are to provide
starter funds for early Translational work. In this portfolio, there are awards
for a pilot pre-clinical study, a pilot eye-tracking communication study, and a
pilot collaboration with a contract research organization (CRO) to help develop
and test new therapeutics faster and move them through the pre-clinical stage
towards clinical studies more efficiently through standardized testing.
MD PhD, Tri-State Rett Syndrome Center,
Montefiore Medical Center, Albert Einstein College of Medicine. Language comprehension and processing in
Rett syndrome: A pilot study of eye tracking
Lee-Way Jin, MD
PhD, Regents Of The University Of
California - Davis Preclinical studies of allopregnanolone, a positive GABAA receptor modulator
Daniela Brunner, PhD, PsychoGenics Inc. PPAR-sparing insulin sensitizers for Rett Syndrome