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.
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
Oak Hill Bio has shared a community letter to the Angelman syndrome community with an important update on the next steps of the development of
We found solace and purpose through FAST. Their relentless pursuit of a cure resonated with us, and we started our fundraising journey with them.
