Voice of Patient Report Submitted to FDA

The Voice of the Patient Report from the Externally-Led Patient-Focused Drug Development (EL-PFDD) Meeting on Angelman Syndrome was submitted to the U.S. Food and Drug Administration (FDA). This is a powerful milestone for the Angelman syndrome community.

Hosted by the ASF and FAST, the EL-PFDD meeting on  April 7, 2025, brought together hundreds of families, caregivers, researchers, clinicians, FDA representatives, and industry partners to spotlight the urgent, unmet needs of people living with Angelman syndrome. This powerful report captures the lived experiences of individuals with Angelman syndrome and their families.

This milestone reflects the strength of the Angelman syndrome community—a community that continues to drive science forward and ensure decision makers have the knowledge needed to bring meaningful treatments to life.

Read the full Voice of the Patient Report.