By uniting researchers, medical professionals, and advocates, we aim to make a lasting impact on the lives of those affected. Through your support, we can accelerate scientific breakthroughs and provide hope to individuals and families facing the challenges of Angelman Syndrome every day.
Following Ultragenyx’s announcement that its Phase 3 Aspire trial of GTX-102 did not meet its primary or key secondary endpoints, the Angelman Syndrome Foundation (ASF)
MavriX Bio announced that the FDA has granted Rare Pediatric Disease Designation (RPDD) to its investigational gene therapy, MVX-220. Rare Pediatric Disease Designation is granted
Ionis has shared an important update with the Angelman syndrome community about the design of CHAMPION, its Phase 3 clinical trial evaluating ION582 (obudanersen). The
“Guided by input from the Angelman community, REVEAL was intentionally designed to evaluate obudanersen across a broad range of people living with Angelman syndrome, reflecting
We found solace and purpose through FAST. Their relentless pursuit of a cure resonated with us, and we started our fundraising journey with them.
