
Ultragenyx GTX-102 Aspire trial: community webinar recording
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)
The FAST Global Search & Rescue Initiative has a vital mission: to identify every individual worldwide living with Angelman Syndrome (AS). By connecting families and individuals with AS to resources, clinical trials, and the latest research, the initiative plays a critical role in advancing potential treatments and a cure.
We are excited to announce that the Search & Rescue program is now fully integrated into the Global Angelman Syndrome Registry. This integration enhances data security, simplifies participation for families, and strengthens our global research efforts.
As part of the integration, there will be a more comprehensive consent process, focusing on clinical trial contact and the inclusion of data in research initiatives.
Integrating the Search & Rescue program with the Global Angelman Syndrome Registry creates a unified, powerful resource for connecting families with clinical trials and the latest research. By participating, families help accelerate the path to effective treatments and, ultimately, a cure.
For more information or to join the Search & Rescue initiative, please visit the Global Angelman Syndrome Registry.

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
We found solace and purpose through FAST. Their relentless pursuit of a cure resonated with us, and we started our fundraising journey with them.