Rugonersen Development Update: First Participant Dosed in the Phase 3 BEACON Trial
Oak Hill Bio shares a community letter marking the first participant dosed in its BEACON study, thanking families and investigators for making this milestone possible.
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.
FAST Brings Angelman Syndrome Community Perspective to FDA Rare Disease Roundtable
FAST joined 15 advocacy groups at the FDA Rare Disease Roundtable this week, representing the Angelman syndrome community as therapies advance from research toward real-world development.
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.
Important update on alogabat, an investigational therapy in development for Angelman syndrome
Roche/Genentech has discontinued its alogabat program for Angelman syndrome. FAST thanks trial participants for their commitment and shares the full letter with additional context.
FAST Leaders to Speak at the CNBC Cures Summit on Patient- and Family-Led Advocacy
FAST joins the inaugural CNBC Cures Summit as the only patient advocacy org on stage, spotlighting how family-led models are accelerating rare disease drug development.
An Update on the ION582 clinical development program including HALOS, REVEAL, and CHAMPION Trials
Ionis shares an update on ION582 development, covering progress across the HALOS, REVEAL, and newly announced CHAMPION trials expanding research into additional AS genotypes.
MavriX Bio Announces First Patient Dosed in ASCEND-AS Trial of MVX-220, Investigational Gene Therapy for Angelman Syndrome
Read the press release
Voice of the Patient Report Submitted: A Powerful Milestone for the Angelman Syndrome Community
The Voice of the Patient Report from the Angelman syndrome EL-PFDD Meeting has been submitted to the FDA, capturing families’ lived experiences to help drive meaningful treatments forward.”
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.