Uncategorized Archives | International Rett Syndrome Foundation

January 12, 2026

Neurogene Announces Recent Achievements and Anticipated 2026 Key Milestones for NGN-401, a Potential Best-in-Class Gene Therapy for Rett Syndrome

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 […]

Your Voice Matters to the FDA & Pharmaceutical Industry

By Dr. Dominique Pichard, IRSF CSO – The patient voice has increasingly become a focus for the Food and Drug Administration (FDA) in the drug development process. Thanks to years […]

FDA Grants Trofinetide Priority Review

ACADIA has shared the exciting news that the US Food and Drug Administration (FDA) has reviewed the documents received as part of the New Drug Application (NDA) for trofinetide, and […]

In-Person Strollathons Are Back!

In September IRSF returns with our in-person Strollathons! These nationwide fundraisers bring together local communities to go ALL IN for treatments and a cure for Rett syndrome. Since 2004, these […]

IRSF Expands Center of Excellence Network

The International Rett Syndrome Foundation is expanding our community’s access to best-in-class care by adding four Rett syndrome clinics to our Center of Excellence network. This expansion helps to fulfill […]

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 […]

Taysha Rett Syndrome Community Update

From IRSF: 2022 is a big year for Rett syndrome: the return of an in-person Family Summit, a successful EL-PFDD meeting to share the voice of the community with the […]

IRSF Sets Up Next Wave of Rett Breakthroughs with $2.3 M Research Investment

Dr. Dominique Pichard, IRSF Chief Science Officer Get ready for the next wave of research targeting treatments and cures for Rett syndrome. IRSF is making it happen. “Because Rett syndrome […]

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