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Ketamine Clinical Trial for Rett Syndrome Launches

March 13, 2019Research

You may have heard about a clinical trial testing ketamine in Rett Syndrome. We’re excited to be leading this important study and really asking in an unbiased way if ketamine can improve the symptoms of Rett Syndrome. Ketamine is an old drug and has been studied in all kinds of…

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On the Shoulders of Giants

January 24, 2019Fundraising, Research

2019 started with the exciting announcement that our 2018 research awards totaled $10 million! We take our hats off to the hundreds of Rett families who put their urgency and passion to work raising funds and to our colleague, Tim Freeman, who leads this impressive effort. Although we personally are…

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Three Stars and
Prouder than Ever

January 17, 2019Fundraising, In The News

I’m proud to say that RSRT once again received a three-star rating from Charity Navigator, the organization that assesses the transparency, financial health, and efficiency of non-profits.  The “top” rating is four stars. You might say to me—Tim, are you sure you should be so proud to have three stars…

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What’s Making MeCP2 Toxic in Duplication Syndrome?

January 14, 2019Research

I’m a post-doc in the lab of Adrian Bird. The lab has historically studied MeCP2 in the context of Rett Syndrome – but now we are using everything we’ve discovered about MeCP2 to also better understand its role in MECP2 Duplication Syndrome (MDS). Professor Bird discovered MeCP2 in the early…

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Rett Superheroes

January 4, 2019Roadmap

We launched Roadmap to a Cure, RSRT’s three-year $33 million strategic research plan, in March of 2017.  We’re now 21 months into the 36-month period, 60% through the timeframe.  I’m excited to report that as of January 1 we’ve raised almost $24 million towards the goal.  That’s 73% of the…

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World-Class Team Takes on Next Gen Gene Therapy

December 13, 2018

RSRT first started funding gene therapy efforts in 2010 with a collaboration between Gail Mandel, PhD and Brian Kaspar, PhD. That collaboration led to our original Gene Therapy Consortium that launched in 2014 with the inclusion of Steve Gray, PhD and Stuart Cobb,PhD. The Consortium worked through numerous challenges involving vector optimization…

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New Method Developed to Study MeCP2 Function in Humans

November 19, 2018Research

We wanted to share with the RSRT community a little about a new method we’re using to gain a better understanding of how the MECP2 protein functions. Our expectation is that this research, which is funded by RSRT and the NIH, will help guide approaches to therapeutics that will hopefully…

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