We’re in This Together
June 12, 2018
Last March we announced an ambitious goal at RSRT to raise $33 million over three years to support our strategic research plan, Roadmap to a Cure. This is no arbitrary goal. Every dollar correlates with a research budget that aggressively moves the needle on potentially curative approaches. I’m excited and…
Meeting of the Minds
June 7, 2018
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…
Unapologetically Going for the Cure
April 30, 2018
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…
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…
Igniting Hope
April 13, 2018
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…
Scientists are thinking the unthinkable: CRISPR might one day reverse devastating brain diseases
April 10, 2018
Monica Coenraads’ daughter has never spoken since she blurted out “duck!” while taking a bath soon before her first birthday, and has never walked. Chelsea lost the ability to hold her sippy cup and stopped responding when Coenraads played “can you touch your nose?” She cannot use her hands, and…
Novartis to Acquire AveXis
April 9, 2018
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…
