Landmark event to raise the voice of the Angelman syndrome community with drug developers and the Food and Drug Administration
August, 1, 2024 – We are excited to announce that on January 29, 2025, the Angelman syndrome community will have a unique opportunity to share our experiences on living with the condition, including our perspectives on treatments for our loved ones, with drug developers, clinicians, and FDA staff at a virtual externally-led patient-focused drug development (EL-PFDD) meeting. (opens in a new window)
Co-hosted by the Foundation for Angelman Syndrome Therapeutics and the Angelman Syndrome Foundation (opens in a new window), Hope in Action: an EL-PFDD Meeting on Angelman Syndrome, is a platform for our community to share insights and priorities directly with the FDA and other stakeholders, ultimately shaping the future of therapy development.
The goals of the half-day meeting are:
- To spotlight what matters most to caregivers on behalf of individuals living with Angelman syndrome;
- To improve understanding of the challenges of living with Angelman syndrome and aspects of the condition the community would most like to address with new treatments;
- To inform drug development programs and related FDA reviews of potential Angelman syndrome treatments.
Learn more and RSVP to join the meeting: the EL-PFDD meeting page