Today, Taysha Gene Therapies announced that a 2nd adult patient has been dosed with TSHA-102 in their REVEAL Phase 1/2 trial of the investigational gene therapy in Canada. They expect to dose the 3rd adult patient and complete enrollment in this low-dose cohort by the end of this year, and plan to dose the first … Read More
Today, Taysha Gene Therapies made several exciting announcements about the progress of their REVEAL Phase 1/2 trial in Canada and the future study of their gene therapy, TSHA-102. To read their full press release, click here. Key Facts: Updates on the first adult patient dosed in the REVEAL Phase 1/2 trial in Canada: TSHA-102 was … Read More
BIG NEWS for our international Rett syndrome community! Today, Acadia Pharmaceuticals announced they have acquired the rights to make trofinetide available for the treatment of Rett syndrome across the globe. READ THE PRESS RELEASE This is an essential starting point in ensuring ALL Rett families have access to this treatment. Acadia already retains the rights … Read More
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