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

Neurogene Announces Locations & Opens Enrollment for US Gene Therapy Clinical Trial

Monday, June 12, 2023

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

Taysha Announces First Patient Dosed with Gene Therapy

Monday, June 5, 2023

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

A First for Rett: FDA Approves Trofinetide for Treatment of Rett Syndrome!

Friday, March 10, 2023

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

IRSF Establishes Research Fund for Males with Rett Syndrome

Tuesday, February 21, 2023

The fund is established thanks to an inaugural gift from the Otis Family in honor of their son, Barrett. The Rett syndrome research landscape has never been more exciting. New clinical trials are underway in gene therapy, and we eagerly await the FDA’s decision about Trofinetide. But we know that families of males with Rett … Read More