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
For Jack: Sonia and Jose Take on the Inca Trail
Before Sonia and her son José set off on Peru’s Inca Trail this September, their guides gave the group two goals: survive first, and finish if you can. For Sonia and José, finishing mattered. They were hiking in honor of Jack, Sonia’s son, who lives with Angelman syndrome, and raising money for research through FAST’s […]
MavriX Bio Announces FDA Rare Pediatric Disease Designation for Investigational MVX-220 for the Treatment of Angelman Syndrome
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Beyond UBE3A: New Conversations About Genotype, Variants, and Future Treatments
At INSYNC-AS 2026, researchers came together to examine how genes beyond UBE3A and differences within UBE3A itself may shape Angelman syndrome and future therapeutic development. Why can Angelman syndrome (AS) look different from one person to another, even when the diagnosis is the same? And could understanding those differences help researchers develop better treatment approaches […]
Skylark Bio Expands Pipeline with First CNS Development Program, SKY-AS, Targeting UBE3A Restoration in Angelman Syndrome
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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
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
Foundation for Angelman Syndrome Therapeutics Announces Agreement with Apertura Gene Therapy for Access to Blood-Brain Barrier-Crossing Capsid
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MavriX Bio Announces First Patient Dosed in ASCEND-AS Trial of MVX-220, Investigational Gene Therapy for Angelman Syndrome
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MavriX Bio Receives FDA Fast Track Designation for MVX-220 for Treatment of Angelman Syndrome
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