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World-Class Team Takes on Next Gen Gene Therapy

December 13, 2018

RSRT first started funding gene therapy efforts in 2010 with a collaboration between Gail Mandel, PhD and Brian Kaspar, PhD. That collaboration led to our original Gene Therapy Consortium that launched in 2014 and also included Steve Gray, PhD and Stuart Cobb,PhD. The Consortium worked through numerous challenges involving vector optimization (the…

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New Method Developed to Study MeCP2 Function in Humans

November 19, 2018Research

We wanted to share with the RSRT community a little about a new method we’re using to gain a better understanding of how the MECP2 protein functions. Our expectation is that this research, which is funded by RSRT and the NIH, will help guide approaches to therapeutics that will hopefully…

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Talk To Your Doctors

November 14, 2018Community

If your daughter (or son) is approaching 18 years old, there are a couple of matters that you must handle. While most people know that when our children turn 18 years of age, they are no longer ours, but they are their own person. For me, that meant working with…

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What Does it Take to Run a Clinical Trial?

November 6, 2018Research

Ever wondered how easy or hard it might be to run a clinical trial, or what researchers have to do to be able to run a study? Here we’ll outline some key differences between an academic study and an FDA-regulated study, and talk about our own ketamine clinical trial planned…

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UPDATE: AVXS-201 Gene Therapy for Rett Status

October 31, 2018Research

The entire Rett community celebrated on May 3 of this year when AveXis formally announced plans to submit the IND application for AVXS-201 for Rett syndrome in late 2018/early 2019. We all then held our breath on May 15 when Novartis purchased AveXis for $8.7 billion, wondering whether Novartis would…

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Moving the Needle

October 29, 2018Research

I have always enjoyed the creative process. In my younger days, I was fond of cooking, which was not always sanctioned as an appropriate activity for a young boy in that era of our society. As I moved forward with my high school education, I had an intense interest in…

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Confessions of a Novice Event Planner

October 23, 2018Fundraising, Success Stories

Let me start by disclosing that I am not an event planner nor am I experienced in the world of fundraising or social advocacy. I am a psychologist, an introvert, a dreamer, a neophyte on social media, and most importantly, I am a mom. Before reaching out to RSRT, I…

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