Rettsyndrome.org reaches milestone of $40 million in research funding

January 09, 2017

(Cincinnati, OH) The International Rett Syndrome Foundation, now doing business as Rettsyndrome.org, announces today that Rettsyndrome.org has reached a milestone in research funding. The leading foundation for Rett research has distributed over $40 million dollars to accelerate treatments for Rett syndrome.

In December 2016, an additional $2M was granted to support four breakthrough human clinical trials for potential pharmacological interventions. Rettsyndrome.org also funded the renewal of two database grants and granted an extension of the Scout Program for drug discovery efforts.

Rettsyndrome.org continues to be the undisputed non-governmental leader in driving clinical research with results. In early 2016, Rettsyndrome.org funded the largest multi-site human clinical trial of Neuren Pharmaceutical's drug trofinetide. This trial completed enrollment in late 2016 and results from the trofinetide drug trial should be available in mid-2017. Phase 2 IGF-1 trial at Boston Children's Hospital has also completed enrollment and all study visits. Results will be available in 2017.

In late 2016, Newron pharmaceuticals began enrollment throughout the US, UK, Italy, and India for their study of Sarizotan, a potential treatment for breathing abnormalities found in Rett syndrome. This is the first pivotal Phase 2/3 clinical trial located at multiple sites.

Rettsyndrome.org's Chief Science Officer, Steve Kaminsky, PhD, shares, "With an investment of over $40 million dollars into understanding, treating and curing Rett syndrome our organization continues to move the bar in helping all of those suffering with Rett syndrome. Our recent investments into clinical trials bring us closer to our goal of changing the lives of those with Rett syndrome NOW. Together with all of our wonderful donors we are bring about changes in this devastating disorder."
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About Rettsyndrome.org

Rettsyndrome.org is the most comprehensive nonprofit organization dedicated to accelerating research of treatments and a cure for Rett syndrome to accelerate full spectrum research to treat and cure Rett syndrome while empowering the community through knowledge and connectivity.

As the world's leading private funder of Rett syndrome research, Rettsyndrome.org has funded over $40M in high-quality, peer-reviewed research grants and programs to date. The organization hosts the largest global gathering of Rett researchers and clinicians to establish research direction for the future. Rettsyndrome.org, a 501(c)3 organization, has earned Charity Navigator's most prestigious 4 star rating year after year. To learn more about our work and Rett syndrome, visit http://www.rettsyndrome.org or call (800) 818-7388 (RETT).

Rettsyndrome.org

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