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.
Co-hosted by the Foundation for Angelman Syndrome Therapeutics and the Angelman Syndrome Foundation, 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.
We need your direct involvement in this initiative. Please join our webinar on Tuesday, August 20 at 9:00pm ET (note: previously scheduled for 8pm ET). We plan to share more information on patient-focused drug development, the Hope in Action meeting format and goals, meeting logistics, and the opportunity to participate in a key community survey in October.