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) and FAST hosted a community webinar with Ultragenyx. The webinar provided an opportunity for the Angelman community to hear from Ultragenyx about the trial results and ask questions. Watch the […]

Thomas’s Fulham raises over £6,500 for FAST UK

On Saturday 12 September 2026, a brilliant team of pupils and parents from Thomas’s Fulham completed the Thames Bridges Trek in support of FAST UK, raising an incredible £6,583 at the time of writing.

MavriX Bio announced that the FDA Rare Pediatric Disease Designation

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 by the FDA to therapies intended to treat serious or life-threatening diseases that primarily affect individuals from birth through 18 years of age and affect fewer than 200,000 people in […]

Ionis shares update on CHAMPION Phase 3 clinical trial

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 study will include children and adults with Angelman syndrome caused by uniparental disomy (UPD) or an imprinting centre defect (ICD). Ionis is actively preparing to begin the CHAMPION study. The […]

Ionis completes enrolment in pivotal cohort of the Phase 3 REVEAL study evaluating obudanersen in Angelman syndrome

“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 the real-world diversity of this condition. We are deeply grateful to the entire Angelman syndrome community, especially the participants and their families, whose partnership made this achievement possible.” – Holly […]

Support FAST UK Every Time You Shop Online – At No Extra Cost

The donations generated from individual purchases may seem small, but when our community comes together, those donations quickly add up. By choosing to shop through easyfundraising, you’re helping to support the research that brings us closer to better treatments and ultimately a cure for Angelman syndrome.