European Commission grants NTI64 Orphan Drug Designation for Rett Syndrome

Neurogene Reports Fourth Quarter and Full Year 2024 Financial Results and Highlights Recent Updates

Clinical Data from Open-Label DAFFODIL™ Study Evaluating Long-term Safety of DAYBUE® (trofinetide) in Patients with Rett Syndrome Published in Med

Taysha Gene Therapies Reports Full-Year 2024 Financial Results and Provides Corporate Update

Acadia Pharmaceuticals Submits Marketing Authorization Application to the European Medicines Agency for Trofinetide for the Treatment of Rett Syndrome

Neurotech Receives US Orphan Drug Designation in Rett Syndrome

The Summit of Hope and Healing: ASCEND 2022 Rett Syndrome National Summit

By Katie Busch, IRSF Program Coordinator –  During the last week of April 2022, the International Rett Syndrome Foundation welcomed more than 500 members of the Rett community to the […]

Accelerating Discoveries in Rett Research: 2022 IRSF Rett Syndrome Scientific Meeting

In April 2022, the International Rett Syndrome Foundation hosted the IRSF Rett Syndrome Scientific Meeting in advance of the foundation’s first in-person family summit in 6 years. The 2-day meeting […]

Alcyone Presents Preclinical Data on Gene Therapy Programs with The Center for Gene Therapy at The Abigail Wexner Research Institute (AWRI) at ASGCT 2022

Taysha Gene Therapies Announces Initiation of Clinical Development of TSHA-102 in Rett Syndrome

Acadia Pharmaceuticals and Stoke Therapeutics Announce Collaboration to Pursue Multiple RNA-based Treatments for Severe and Rare Genetic Neurodevelopmental Diseases

Acadia Pharmaceuticals Announces Positive Top-line Results from the Pivotal Phase 3 Lavender Trial of Trofinetide in Rett Syndrome