Patient and care partner perspectives on safety considerations for approved gene therapy treatments for rare diseases (from Catapult's regulatory round-up for November 2024)

The FDA Center for Biologics Evaluation and Research (CBER) hosted a public patient listening meeting on the 20th September 2024 and opened a docket to better understand patient and care partner perspectives on safety considerations and long term follow-up for approved gene therapy treatments for rare diseases. The video recording of this meeting is available here. Published on the 3rd of december 2024. Read the entire Regulatory Round-up – November 2024 here.