2016: A Year in Review
July 19, 2017
Thanks to our almost 9,000 donors we raised a record amount in 2016. We’re putting that money to work on behalf of everyone struggling with Rett Syndrome and related MECP2 disorders. We invite you to view our infographic. Thank you to all our supporters! You can also download the infographic here. …
What About Us?
July 12, 2017
It’s been almost two decades since the discovery that alterations in MECP2 cause Rett Syndrome. However, the diagnosis of Rett remains a clinical one based on a child’s symptoms and history. While a positive MECP2 genetic test confirms the diagnosis, it is not required. This creates a situation where individuals can…
Finding the Words
June 26, 2017
I told my daughter, Eleanor, the news. I had just put her in bed for the night. Her arms had stopped moving and she was breathing quietly. She stared into my eyes with that intense look that only the parents of a daughter with Rett Syndrome can understand—intelligent, lucid, expressive…
Precision Medicine
June 19, 2017
Mutations in a single gene, MECP2, can be identified in the vast majority of individuals with Rett Syndrome, yet each person is unique. As in any disease, treatments such as seizure or anxiety medications can be very successful for some individuals but not for others, even for those with the…
AveXis Announcement – Delivering on Roadmap to a Cure
June 8, 2017
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…
Gene Therapy Consortium Reports on Progress
May 15, 2017
We are excited to report to the Rett Syndrome community and to everyone who cares about an afflicted family on recent progress made by RSRT’s Gene Therapy Consortium. As you may know, Rett Syndrome is caused by spontaneous mutations in the MECP2 gene that prevents the production of healthy MeCP2…
Gene Therapy Primer
May 10, 2017
Gene therapy is the lead program of our three-year strategic plan, Roadmap to a Cure. This potential one-time treatment, which targets Rett at its very root, has produced dramatic results in mouse models of the disease, more so, by far, than any other drug treatment studied thus far. Let’s review…
