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
By Samantha Brant, Family & Community Engagement Manager, and mom to Macy As I returned home from this year’s RettAway at Morgan’s Inspiration Island, my heart swelled with not only pride but warmth. I attended the last RettAway in 2017 as simply a parent to a 3-year-old little peanut, Macy. And I took so much … 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
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