RSAM, Rettsyndrome.org renew commitment to defeat Rett syndrome

Published on August 7, 2014 at 1:20 AM · No Comments

Rett Syndrome Association of Massachusetts (RSAM) and Rettsyndrome.org are thrilled to announce today their renewed commitment to work together to defeat Rett syndrome. A generous $150,000 donation from RSAM will support a Rettsyndrome.org approved grant to Dr. Michela Fagiolini's project at the Boston Children's Hospital: "Assessing NMDA receptor (NMDAr) modulators to ameliorate cortical regression in Rett syndrome".

Rajat Shah, Rettsyndrome.org Chairman of the Board, commented, "Rett Syndrome Association of Massachusetts has been a strong partner of Rettsyndrome.org for many years now. We are grateful and honored to grow our partnership in this significant way and take our collaboration to the next level. It will allow us to be more effective in reaching our common goal, developing treatments for our children, faster."

The Fagiolini laboratory at Boston Children's Hospital will conduct a preclinical study in a Rett mouse model, testing two very promising modulators of the NMDA (N-methyl-D-aspartate) receptor. These receptors are important for learning and memory and are somewhat impaired in Rett syndrome. The study will target the NMDA receptor with two different drugs, FDA approved ketamine and a new inhibitor of the NMDA receptor GLYX-13. They will evaluate the safety, pharmacokinetic activity, and efficacy of each drug. If the drugs are deemed safe and efficacious as positive and robust outcomes in the preclinical study, they will ready a potential clinical trial design with the goal of moving the compounds quickly to human clinical studies and evaluate these NMDA receptor modulators as potential treatments in girls diagnosed with Rett syndrome.

Jane Joyce, on behalf of RSAM, said, "We are incredibly grateful to the RSAM 2014 Boston Marathon Team Rett runners. Not only did the runners endure months of training in preparation for the marathon, but they committed to raising significant funds for Rett syndrome research. Our team of 26 runners, which included parents, aunts, cousins and friends of girls with Rett syndrome, went above and beyond this year. We are so thankful for their dedication and support. RSAM is pleased to be able to fully-fund Dr. Fagiolini's promising line of science and to continue our relationship with Rettsyndrome.org. "

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