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Mecasermin (rh-IGF-1) treatment for Rett Syndrome is safe and well-tolerated
Date:3/10/2014

nd breathing, and further investigate other potential improvements. The Phase 2 trial will include 30 children with Rett syndrome between the ages of 2 to 10 years old who are in the "stable" stage of the disease. The Phase 2 trial is currently at its midpoint and, if successful, it will be followed by a larger replication Phase 3 study. The latter is a requirement for FDA approval of IGF-1 indication for RTT. Altogether, these IGF-1 trials will open the path for the testing of additional potential treatments for RTT.

IRSF anticipates that Rett syndrome research momentum will accelerate towards more additional clinical trials in the near future. Whether support for expansion of the current trials to Phase 3 or for implementation of Phase 1 trials for new drug candidates will be needed, IRSF is at the forefront of clinical research for Rett syndrome. Dr. Steven Kaminsky, IRSF Chief Science Officer said, "These clinical research studies are critical to developing pharmacologic treatments for the patients with Rett syndrome now. These studies combined with forward looking abilitation therapies will provide avenues to change the quality of life for those suffering with Rett syndrome and their families."

"With hope to quickly bring treatments to our girls, IRSF is actively working on a campaign to fund additional human clinical trials including the possible expansion of current studies to Phase 3," said Rajat Shah, IRSF Chairman of the Board. "Thank you to all the girls, families, and donors who supported these Phase 1 and Phase 2 clinical trials and IRSF."


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Contact: Steve Kaminsky, Ph.D.
skaminsky@rettsyndrome.org
301-961-1559
International Rett Syndrome Foundation
Source:Eurekalert

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