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
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
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