
RSRT
@rsrt
The Rett Syndrome Research Trust: Our singular focus is a cure for Rett syndrome.
DONATE: reverserett.org/donate/
ID: 25153715
http://www.reverserett.org 18-03-2009 21:01:36
1,1K Tweet
2,2K Followers
305 Following

Learn more about #genetherapy, the value to #patients & policy updates needed to #EnsureAccess. Register for Institute for Gene Therapies event "Realizing the Promise of Gene Therapy: Ensuring Access" on 6/26 to hear from patient advocates and IGT leaders. forms.gle/u6hFmagnCeaLQs…



Brief update from Taysha Gene Therapies on Patient #1 in their Rett gene therapy trial. ir.tayshagtx.com/news-releases/…


FDA has just approved a gene therapy for hemophilia A. BioMarin’s president of R&D, Henry Fuchs, sums it up as follows: “The learning here was it doesn’t work in everybody. It doesn’t last forever. Our view is it works in enough people and long enough.” endpts.com/biomarins-hemo…





RSRT is laser-focused on genetic medicines. Gene therapy is a genetic medicine that delivers healthy genes to compensate for mutated ones. Learn more about this approach with this short explainer from the American Society of Gene and Cell Therapy. patienteducation.asgct.org/gene-therapy-1…








