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Research

Research
You may have heard about a clinical trial testing ketamine in Rett Syndrome. We’re excited to ...
Research
2019 started with the exciting announcement that our 2018 research awards totaled $10 million! ...
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I’m a post-doc in the lab of Adrian Bird. The lab has historically studied MeCP2 in the ...
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RSRT first started funding gene therapy efforts in 2010 with a collaboration between Gail ...
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We wanted to share with the RSRT community a little about a new method we’re using to gain a ...
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Ever wondered how easy or hard it might be to run a clinical trial, or what researchers have to ...
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The entire Rett community celebrated on May 3 of this year when AveXis formally announced plans ...
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I have always enjoyed the creative process. In my younger days, I was fond of cooking, which ...
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The DNA double-helix was first postulated to be the building blocks of life by Watson and Crick ...
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Most scientific meetings strive to inform the audience on the state of the field by presenting ...
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A recent article entitled “A perspective on “cure” for Rett syndrome” authored by a ...
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As the third blog in a series introducing drug development, I am thrilled to be discussing ...
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Monica Coenraads’ daughter has never spoken since she blurted out “duck!” while taking a ...
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AveXis announced today that it has entered into an agreement to be acquired by the ...
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The AveXis announcement ushers in a new and exciting era of clinical trials that attack the ...
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Peer review, it’s a term you’ve probably heard numerous times. But what exactly is it? As ...
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Our recent announcement that AveXis is planning to initiate a gene therapy clinical trial ...
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AveXis Reports on the Rett Syndrome Gene Therapy Program – Clinical Trial Likely to Start ...
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Andrew Carnegie, the 19th century business magnate and philanthropist, once said, “It is more ...
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First, what is the FDA and what do they do? Many people assume that the FDA develops ...
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I’m a stickler for accuracy. I always have been. So I confess to being a tad uncomfortable ...
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A lot has happened in the six months since publication of our Gene Therapy Primer. Notably, two ...
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It’s been an exciting several weeks for the field of RNA and DNA editing. Three days after ...
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Until we know what form the Rett cure will take it’s critical to pursue multiple strategies ...