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
Coming to your mailbox this October – our 2022 Rett Gazette is here! Find out how we are continuing to accelerate vital, innovative research while helping families thrive, no matter where they are on their Rett Journey. This year’s issue highlights how a world without Rett syndrome requires a powerful synergy between families and research, … Read More
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 they have determined that the application is complete and can be filed. They also determined that trofinetide qualifies for Priority Review. The Prescription Drug User … 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
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