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The Future is Now

August 7, 2018Research

The DNA double-helix was first postulated to be the building blocks of life by Watson and Crick in a seminal paper in 1953. Their discovery spawned a revolution in molecular biology in the 65 years that have followed, and led to the understanding of human biology, human disease and our…

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Meeting of the Minds

June 7, 2018Research

Most scientific meetings strive to inform the audience on the state of the field by presenting data in a consolidated manner, maximizing speaker time often at the expense of discussion time. Separating clinical and scientific sessions is common. This year, when planning our Investigator Meeting in Boston on May 21…

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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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Novartis to Acquire AveXis

April 9, 2018Research

AveXis announced today that it has entered into an agreement to be acquired by the pharmaceutical company, Novartis. Highlights of the announcement follow: The proposed purchase includes AveXis’ lead compound for SMA as well as the pipeline programs for Rett Syndrome and ALS. The Rett program, AVXS-201, continues full steam…

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Drug Development Is Cheap And Easy, Said No One Ever

March 15, 2018Research

The AveXis announcement ushers in a new and exciting era of clinical trials that attack the root cause of Rett Syndrome. Without a lot of precedent with gene therapy just yet, it’s hard to know what FDA will require and what the development timelines will look like. In my last…

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Making It Count

March 12, 2018Fundraising, Research

I’ve blogged about how RSRT solicits and reviews proposals. But what happens once an award is made? For most funding organizations making the award is the last step in the process. The MO (modus operandi) is “congratulations on the award, good luck and send us a report in a year…

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