Category

Research

2nd Adult Patient Dosed in Taysha Gene Therapies Study of TSHA-102

Tuesday, September 26, 2023

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

Acadia Pharmaceuticals Acquires International Rights to Trofinetide

Thursday, July 13, 2023

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

IRSF Welcomes Three New Members to its Board of Directors

Monday, July 10, 2023

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

Clinical Trial Updates from Taysha & Anavex

Wednesday, June 28, 2023

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