Research Archives - Page 5 of 8 - International Rett Syndrome Foundation

IRSF Launches Clinical Trial Committee to Support the Development of Rett Syndrome Treatments

Tuesday, May 31, 2022

The Clinical Trial Committee includes 14 leading U.S. Rett syndrome specialists CINCINNATI – (May 31, 2022) – The International Rett Syndrome Foundation (IRSF) announced today that its Clinical Trial Committee (CTC) has launched, having completed its first protocol review, and is available as a resource to drug developers and pharmaceutical companies planning clinical trials to … Read More

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

Thursday, May 26, 2022

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 brought together more than 150 clinical, academic, and industry experts from around the world, all working towards the common goal of developing effective treatments and … Read More

Taysha Rett Syndrome Community Update

Wednesday, March 30, 2022

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 FDA, ACADIA will submit a new drug application to the FDA to request approval of trofinetide as the first medication specifically targeting Rett syndrome, and … Read More

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

Wednesday, December 22, 2021

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 is a rare disorder, our Foundation’s investments strongly influence the direction and pace of Rett research, as well as the potential treatment options that come … Read More

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

Monday, December 6, 2021

– Trofinetide met co-primary efficacy endpoints demonstrating statistically significant improvement over placebo in the Rett Syndrome Behaviour Questionnaire (RSBQ) (p=0.0175) and the Clinical Global Impression of Improvement (CGI-I) (p=0.0030) – Trofinetide met key secondary endpoint demonstrating statistically significant improvement over placebo in CSBS-DP-IT–Social (p=0.0064), caregiver scale of ability to communicate – Pre-New Drug Application meeting … Read More