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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 ...
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We’ve known for almost two decades that mutations in the MECP2 gene and its corresponding ...
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It’s been almost two decades since the discovery that alterations in MECP2 cause Rett ...
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Mutations in a single gene, MECP2, can be identified in the vast majority of individuals with ...
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The recent announcement that the gene therapy biotech company, AveXis, is advancing the program ...
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We are excited to report to the Rett Syndrome community and to everyone who cares about an ...
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Gene therapy is the lead program of our three-year strategic plan, Roadmap to a Cure. This ...
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RSRT has announced Roadmap to a Cure, a 3-year research plan featuring gene therapy as the lead ...
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Background In the early 1980s, few drugs were being developed for rare diseases as the small ...
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Rett syndrome-causing missense mutations cluster in two regions of MECP2. One of these regions ...
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We’ve tried to anticipate some of the questions that families will have about Roadmap to a ...
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RSRT was launched in 2008, in the wake of Adrian Bird’s remarkable reversal experiment. Our ...