Nav: Home

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."
-end-
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

Related Rett Syndrome Articles:

A dietary supplement improves skills of an atypical Rett syndrome patient
Administration of the amino acid L-serine, a dietary supplement, contributes to the improvement of the communicative and motor skills of a patient with a mutation that alters glutamate receptors.
A new drug shows preclinical efficacy in Rett syndrome
A new article published in the Cell Reports describes how a new drug is able to reduce the symptoms and activate the dormant neurons characteristic of Rett Syndrome in preclinical models.
X chromosome reactivation could treat Rett syndrome, other X-linked disorders
A study from a team of Massachusetts General Hospital investigators points toward a potential strategy for treating X-linked disorders -- those caused by mutations in the X chromosome -- in females.
Discovery fuels hope for Rett syndrome treatment
Vanderbilt University researchers have relieved symptoms of Rett syndrome in a mouse model with a small molecule that works like the dimmer switch in an electrical circuit.
Drug improves brain performance in Rett syndrome mice
A brain penetrant drug -- a small-molecule mimetic of BDNF, or brain derived neurotrophic factor -- is able to improve brain performance in Rett syndrome mice -- specifically synaptic plasticity in the hippocampus and object location memory.
More Rett Syndrome News and Rett Syndrome Current Events

Best Science Podcasts 2019

We have hand picked the best science podcasts for 2019. Sit back and enjoy new science podcasts updated daily from your favorite science news services and scientists.
Now Playing: TED Radio Hour

Rethinking Anger
Anger is universal and complex: it can be quiet, festering, justified, vengeful, and destructive. This hour, TED speakers explore the many sides of anger, why we need it, and who's allowed to feel it. Guests include psychologists Ryan Martin and Russell Kolts, writer Soraya Chemaly, former talk radio host Lisa Fritsch, and business professor Dan Moshavi.
Now Playing: Science for the People

#537 Science Journalism, Hold the Hype
Everyone's seen a piece of science getting over-exaggerated in the media. Most people would be quick to blame journalists and big media for getting in wrong. In many cases, you'd be right. But there's other sources of hype in science journalism. and one of them can be found in the humble, and little-known press release. We're talking with Chris Chambers about doing science about science journalism, and where the hype creeps in. Related links: The association between exaggeration in health related science news and academic press releases: retrospective observational study Claims of causality in health news: a randomised trial This...