The International Rett Syndrome Foundation (IRSF), a mission-driven advocacy foundation focused on funding research and supporting families impacted by Rett syndrome, today announced the expansion of its Board of Directors. Mary Engel, Laura Stoppel, and Steve Wood have been appointed to the IRSF Board, effective July 1, 2023. “We are delighted to welcome these new … Read More
We are excited to share news from two pharmaceutical companies working on potential treatments and cures for Rett syndrome. Taysha Gene Therapies Taysha Gene Therapies shared that they are encouraged by the initial clinical observations of the first adult patient dosed in their TSHA-102 REVEAL Phase 1/2 gene therapy trial in Canada. Further clinical updates … Read More
We’re excited to share that today Neurogene announced the locations and began enrolling for the first U.S. Phase 1/2 clinical trial of their investigational gene therapy, NGN-401. Enrollment at Texas Children’s Hospital begins today, with enrollment at Children’s Hospital Colorado and Boston Children’s Hospital anticipated to open soon. The trial will enroll five (5) females aged … Read More
A First in Rett Syndrome! Taysha Gene Therapies announced today that the first Rett syndrome patient has been dosed with the investigational gene therapy, TSHA-102, in a clinical trial for females 18 and older being run out of Montreal, Canada. Read their press release and full community update below. ———————- June 5, 2023 Dear Rett … Read More
This first-ever treatment is approved for individuals with Rett syndrome. (Updated March 19, 2023) On March 10, 2023, Acadia Pharmaceuticals announced that their investigational drug, Trofinetide, has been approved by the U.S. Food and Drug Administration (FDA) for the treatment of Rett syndrome. The drug, marketed under the name DAYBUE™, is expected to be commercially … Read More