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
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
Ionis completes enrollment in pivotal cohort of the Phase 3 REVEAL study evaluating obudanersen in Angelman syndrome
Ionis completes enrollment for Cohort 1 of the pivotal Phase 3 REVEAL study evaluating obudanersen for Angelman syndrome. Cohort 2 screening continues for ages 18–50.
Update on Rugonersen Development and Planned Phase 3 Study (BEACON)
Oak Hill Bio shared an update on rugonersen’s development, preparing to launch its Phase 3 study in mid-2026. Eligibility details are now available on clinicaltrials.gov.
FAST × ASA Angelman Syndrome Clinical Trials Webinar: Europe/UK Sites, Eligibility, and How to Join Trials
FAST and ASA co-hosted a UK/Europe session with Ultragenyx and Ionis, giving families clear updates on ASO trial programs ASPIRE and REVEAL, tailored to UK/EU time zones.
Oak Hill Bio to continue the development of rugonersen, an investigational ASO previously owned by Roche.
Oak Hill Bio has entered into an exclusive global licensing agreement with Roche to acquire and advance rugonersen, a Phase 3-ready investigational ASO treatment for Angelman syndrome.
IONIS REVEAL Phase 3 Clinical Trial Update
The next chapter for ION582 is here: Ionis’ Phase 3 REVEAL trial is now listed, marking an important step toward a potential treatment for Angelman syndrome. Join FAST, ASF, and Ionis to learn what comes next.
Ultragenyx Announces First Patient(s) Dosed in Pivotal Phase 3 Aspire Study Evaluating GTX-102 in Angelman Syndrome
Ultragenyx announces the first patients dosed in its pivotal Phase 3 ASPIRE study evaluating GTX-102 for Angelman syndrome, a major step toward potential treatment.
Ultragenyx Community Update October 2024
The Phase 3 Efficacy and Safety Study of GTX-102 (ASO) in Pediatrics, sponsored by Ultragenyx, is now listed on ClinicalTrials.gov. Interested in learning more? Watch the Ultragenyx Community Webinar