Dear Friends,


We would like to take a moment to thank you and to celebrate your impact in accelerating research toward treatments and a cure for Rett syndrome. Without your continued support, our recent research breakthroughs would not be possible.

In our Spring/Summer 2017 Rett Gazette, we highlight our major research advances, including three compounds in various stages of human clinical trials. As trofinetide enters into a Phase 3 trial, Rettsyndrome.org is one step closer to bringing a prescribeable treatment to those impacted by Rett syndrome. 


In a relatively short time, trofinetide has shown success in two clinical trials, yet our work is not finished. Trofinetide is just one treatment with potential. Rett syndrome is an incredibly complex brain disorder and multiple approaches will be needed in order to treat or cure all of those impacted. No two individuals with Rett syndrome are the same, even if they share the same MECP2 mutation; so it is unlikely there will be one treatment approach. In addition to the complexity of the brain disorder, we have to think about the age of those affected at the time treatment options become available. While pharmacological interventions are critical, both children and adults will also need neuro-habilitative therapies, such as occupational and physical therapy, as part of their treatment. Therefore, we need to keep pursuing our full-spectrum research strategy. 


The infographic below of the treatment pipeline for Rett syndrome helps explain that Rettsyndrome.org is pursuing all options; from gene therapy to pharmacological interventions. We hope you realize that research takes time.  While this work continues, it is important to celebrate every accomplishment along the path.


We are extremely proud to see the progress and the impact that we are all making together. We need your support to ensure continued success in finding treatments and a cure. With both your time and financial contributions, you are improving the lives of every person affected by Rett syndrome.


Gordy Rich                                                        Steve Kaminsky

Chief Operating Officer                                 Chief Science Officer

 

 

 



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