Uncategorized Archives | International Rett Syndrome Foundation

December 12, 2025

Acadia Pharmaceuticals Announces FDA Approval of DAYBUE® STIX (trofinetide) for Oral Solution, a New Powder Formulation of Trofinetide for the Treatment of Rett Syndrome

Taysha Gene Therapies Updates Community on Positive Initial Findings and Expansion of TSHA-102 Clinical Trial

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 […]

Acadia Pharmaceuticals Acquires International Rights to Trofinetide

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. […]

RettAway Reflections

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 […]

Clinical Trial Updates from Taysha & Anavex

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 […]

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

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 […]

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

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 […]

Neurogene Announces First Rett Syndrome Gene Therapy Trial in U.S.

We’re excited to share that Neurogene announced today that the FDA approved their Investigational New Drug Application (IND) to launch a clinical trial for their gene therapy treatment NGN-401 in […]

Flu, COVID, & RSV: A Message to our Rett Community

By Dr. Tim Benke, IRSF Medical Advisor As many of you know, the United States is currently facing the triple impact of flu, RSV, and COVID-19. These respiratory illnesses are […]

Taysha Begins Recruitment for Gene Therapy Trial

Next Huge Milestone for Rett The last 12 months have been an unprecedented time for Rett syndrome. In December 2021, Acadia Pharmaceuticals reported that the first (and only) Phase 3 […]

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