Category

Family Empowerment

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

In-Person Strollathons Are Back!

Tuesday, July 26, 2022

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 family-friendly events powered by dedicated volunteers, sponsors, donors, and Rett families have raised more than $15M to advance IRSF’s mission to empower families and accelerate … Read More

IRSF Expands Center of Excellence Network

Wednesday, July 20, 2022

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 IRSF’s commitment that all families living with Rett syndrome in the U.S. have ready access to a Rett syndrome expert. “When we see care deserts, … Read More

Acadia Community Update on Trofinetide

Wednesday, July 20, 2022

July 18, 2022 Dear Rett Community, Acadia is pleased to announce the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for trofinetide for the treatment of Rett syndrome in adults and pediatric patients 2 years of age and older. The full text of the Acadia press release can … Read More

It’s Giving Tuesday - Go ALL IN for Rett!
It’s Giving Tuesday - Go ALL IN for Rett!

Go All In For Giving Tuesday