Oct 8, 2026: Ultragenyx Community Webinar
On October 8, 2026, ASF and FAST hosted a webinar with Ultragenyx after the announcement that their phase 3 trial for GTX-102 (aspire) did not meet primary or key secondary endpoints. The webinar recording is now available: https://youtu.be/Jl43UtOVFm4
MavriX Bio Announces FDA Rare Pediatric Disease Designation for Investigational MVX-220 for the Treatment of Angelman Syndrome
Read the press release
Skylark Bio Expands Pipeline with First CNS Development Program, SKY-AS, Targeting UBE3A Restoration in Angelman Syndrome
Read press release
GTX-102 Phase 3 Results: What We Know, What We’re Still Learning, and Next Steps
FAST’s CSO, Allyson Berent, addresses the Aspire trial results in a candid letter, exploring what the data mean, what questions remain, and how the community will move forward together.
Ultragenyx shares that GTX-102 trials will officially close on Oct 30, 2026.
Read the letter from Ultragenyx to the community: Angelman-Syndrome-Community-Update-9.16
Ionis Pharmaceuticals: A letter to the Angelman syndrome community.
Ionis addresses community questions following the ASPIRE trial results, reaffirming its commitment to obudanersen (ION582) through the HALOS, REVEAL, and CHAMPION trials
Oak Hill Bio: A Message to the Angelman Syndrome Community
Following the recent announcement on the outcome of the phase 3 Aspire trial of GTX-102, Oak Hill Bio has shared a message about their commitment to evaluate rugonersen in their ongoing Phase 3 BEACON trial. Read the letter here
Update on CHAMPION, Phase 3 clinical trial of ION582 (obudanersen)
Ionis shares an update on CHAMPION, its Phase 3 trial for ION582 (obudanersen) in Angelman syndrome due to UPD or ICD, with eligibility details now on clinicaltrials.gov.
Ultragenyx Announces Phase 3 Aspire results in Angelman Syndrome
The Phase 3 ASPIRE study for GTX-102 (apazunersen) did not meet its primary or key secondary endpoints. FAST expresses gratitude to participating families and awaits further details from Ultragenyx.
Foundation for Angelman Syndrome Therapeutics Announces Agreement with Apertura Gene Therapy for Access to Blood-Brain Barrier-Crossing Capsid
Read the press release