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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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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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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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What Does it Take to Run a Clinical Trial?

November 6, 2018Research

Ever wondered how easy or hard it might be to run a clinical trial, or what researchers have to do to be able to run a study? Here we’ll outline some key differences between an academic study and an FDA-regulated study, and talk about our own ketamine clinical trial planned…

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UPDATE: AVXS-201 Gene Therapy for Rett Status

October 31, 2018Research

The entire Rett community celebrated on May 3 of this year when AveXis formally announced plans to submit the IND application for AVXS-201 for Rett syndrome in late 2018/early 2019. We all then held our breath on May 15 when Novartis purchased AveXis for $8.7 billion, wondering whether Novartis would…

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Moving the Needle

October 29, 2018Research

I have always enjoyed the creative process. In my younger days, I was fond of cooking, which was not always sanctioned as an appropriate activity for a young boy in that era of our society. As I moved forward with my high school education, I had an intense interest in…

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