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Unapologetically Going for the Cure

April 30, 2018Research, Roadmap

A recent article entitled “A perspective on “cure” for Rett syndrome” authored by a British clinician, Angus Clarke and Ana Paula Abdala Sheikh has sparked differing reactions from Rett families as well as several Rett organizations. Our partners in the UK, Reverse Rett, wrote an open letter to the authors…

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Funding Collaboration Not Competition

February 26, 2018Research, Roadmap

Andrew Carnegie, the 19th century business magnate and philanthropist, once said, “It is more difficult to give money away intelligently than to earn it in the first place”. I wholeheartedly agree and here’s why. Making wise and effective funding decisions requires a rigorous process and unwavering attention on multiple fronts:…

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Roadmap Update: Jaenisch Lab Award

September 20, 2017Research, Roadmap

In March of this year we announced our strategic research plan, Roadmap to a Cure, the cornerstone of which is four therapeutic approaches that we are undertaking in pursuit of a cure for Rett: gene therapy, MECP2 Reactivation, RNA editing/splicing, and protein replacement. Today’s announcement focuses on MECP2 Reactivation. A…

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AveXis Announcement – Delivering on Roadmap to a Cure

June 8, 2017Research, Roadmap

The recent announcement that the gene therapy biotech company, AveXis, is advancing the program generated by RSRT’s Gene Therapy Consortium is the most exciting news since Adrian Bird’s reversal paper a decade ago. First and foremost the future gene therapy clinical trial will be the first ever that addresses the…

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