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.
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.
FAST at ASGCT 2026
FAST leadership takes the stage at ASGCT’s Annual Meeting, driving key conversations on gene therapy, clinical collaboration, and patient advocacy shaping Angelman syndrome research.
AS Natural History Study Celebrates 20 Years!
Celebrating 20 years of the Angelman Syndrome Natural History Study—decades of data, driven by families and researchers, that continues to fuel discovery and progress.
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.
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.
Turning Possibility Into Progress: The Rush F.A.S.T. Center for Translational Research Opens in Chicago
FAST and Rush University Medical Center opened a $5 million translational research center, a groundbreaking hub built to accelerate Angelman syndrome trials and train specialists.
Oak Hill Bio Announces Publication of Rugonersen Phase 1 Study Results in Nature Medicine
Oak Hill Bio’s Phase 1 TANGELO study results, published in Nature Medicine, show improvements in brain activity and development for children with Angelman syndrome on rugonersen.
Ultragenyx Receives Breakthrough Therapy Designation for GTX-102 in Angelman Syndrome
Ultragenyx’s GTX-102 (apazunersen) has received FDA Breakthrough Therapy Designation for Angelman syndrome, marking a key milestone in its path toward potential approval.
MavriX Bio Announces FDA Clearance of IND Application to Initiate First-in-Human Study of Gene Therapy for Angelman Syndrome
The FDA has cleared MavriX Bio’s IND application for MVX-220, an investigational AAV gene therapy for Angelman syndrome.