RSAM, Rettsyndrome.org renew commitment to jointly defeat Rett syndrome

The Rett Syndrome Association of Massachusetts (RSAM) and Rettsyndrome.org announce today their renewed commitment to work together to defeat Rett syndrome. A generous $85,000 donation from RSAM will support an ongoing clinical trial for Rettsyndrome.org at Boston Children's Hospital (BCH). The trial is funded by a Rettsyndrome.org ANGEL (Advanced Neurotherapeutic Grant of Excellence) grant to Dr. Walter Kaufmann for a Phase 2b clinical study of IGF-1 (mescaserim) for treatment of Rett syndrome and development of Rett-specific novel biomarkers of cortical and autonomic function.

Rajat Shah, Rettsyndrome.org's Chairman of the Board, commented, "The partnership between Rettsyndrome.org and the Rett Syndrome Association of Massachusetts has been strong for many years. We are grateful for their continued support and especially for their commitment to the Phase 2b clinical study of IGF-1. Working together will allow us to be more effective in reaching our common goal, developing treatments for our children faster."

This year Rettsyndrome.org approved extension of the ANGEL award to BCH and Dr. Kaufmann with additional funding of $439,000 approved for FY2015. The extended study was recommended by the Food & Drug Administration (FDA). The FDA's suggestion is that additional subjects as well as an additional trial site are needed to make this a pivotal trial that will demonstrate efficacy. If IGF-1 is shown efficacious in a pivotal trial, we have a better case to gain a drug approval for a new indication, in this case IGF-1 for the treatment of Rett syndrome. The recommendation from the FDA was well received, and BCH and Dr. Kaufmann are working towards opening the second site at Greenwood Genetic Center, where Dr. Kaufmann will move to later this year. BCH and Dr. Scott Pomeroy, Chairman of the Department of Neurology, will continue the clinical trial and accept additional subjects to the study. Rettsyndrome.org and RSAM are supportive of BCH and its leadership throughout the upcoming transition and hopeful the trial will be pivotal for Rett syndrome.

Jane Joyce, on behalf of the Rett Syndrome Association of Massachusetts, said, "We are beyond grateful to the RSAM 2015 Boston Marathon Team Rett runners. Although our team was much smaller this year with only eight runners, our athletes went above and beyond in their fundraising efforts! Not only did the runners endure months of training in some of the snowiest weather ever seen in Boston, they left no stone unturned in their quest to fundraise and raise awareness for Rett syndrome research. We are so thankful for their dedication and support. RSAM is pleased to be able to continue to fund the promising IGF-1 trial at Boston Children's Hospital and to continue our relationship with Rettsyndrome.org. "