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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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Preparing for a Clinical Trial

April 27, 2018

As the third blog in a series introducing drug development, I am thrilled to be discussing participation in a clinical trial. Clinical trials are the only way to get new treatments to market and choosing whether or not to participate in a trial is a significant decision. As you can…

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Igniting Hope

April 13, 2018Fundraising, Success Stories

The days and months after we received the Rett Syndrome diagnosis for our young daughter, Natalie, were the darkest of our lives. There were times that the grief felt insurmountable, that we’d spend the rest of our lives in this shroud of hopelessness. We kept this mostly to ourselves, save…

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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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