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 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
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
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
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 ASCEND 2022 Rett Syndrome National Summit. Held in Nashville, Tennessee, at the Gaylord Opryland Resort & Convention Center, the sold-out summit marked the first in-person … Read More