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

Monday, January 23, 2023

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 2023 in pediatric females. This is the second gene therapy clinical study in Rett syndrome currently underway and the very first gene therapy clinical trial … Read More

IRSF Receives $500,000 Donation from Vezbi Super App

Tuesday, January 3, 2023

International Rett Syndrome Foundation (IRSF) is proud to announce that it is the recipient of a $500,000 donation from the Vezbi Super App as part of its Project Seva. Along with the donation, Vezbi will be creating custom Micro-apps and Communities for use by IRSF and the community of families impacted by Rett syndrome that … Read More

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

Thursday, December 8, 2022

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 currently circulating at levels higher than usual for this time of year, creating a surge of patients seeking care in clinical offices, emergency rooms, and … Read More

Taysha Begins Recruitment for Gene Therapy Trial

Thursday, November 17, 2022

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 clinical trial in Rett syndrome had positive results. As hopefully many of you know, Acadia submitted a New Drug Application to the FDA for approval … Read More

Your Voice Matters to the FDA & Pharmaceutical Industry

Thursday, October 13, 2022

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 of advocacy from patients and caregivers, the FDA has developed mechanisms to hear the patient voice and has encouraged pharmaceutical companies to increasingly include the … Read More