The APBD Research Foundation recently participated in the FDA’s Listening Session on “Patient and Care Partner Perspectives on Early Enrollment into Gene Therapy Clinical Trials for Rare Diseases.”
Becca Reef, MS, CGC, our Scientific Coordinator, spoke on behalf of the APBD community at the 2:11:36 timestamp, sharing insights on the challenges of trial enrollment during early stages of disease progression.
👉 Watch Becca’s 4-Minute Presentation here
👉 Access the Summary Slide here
👉 Add your own perspective to the open FDA docket here (through 02/03/2025)
