Dream Big: One Family’s Journey from the Altar to the Start Line
Thomas and Johanna Kennedy turned their daughter Anabella’s Angelman syndrome diagnosis into a movement—weaving advocacy into their wedding and now running the Dublin Marathon for a cure.
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.
46 Days. 14 States. 2,725 Miles. One Reason: Briar.
Charlie Nuck rode from Maine to Key West—alone and self-contained—raising over $65,000 for Angelman syndrome research in honor of his niece Briar, one conversation and one mile at a time.
Spring Advocacy Update: Educating, Advocating, and Building Momentum
A productive spring for Hope in Action: all Angelman syndrome priorities included in House FY27 appropriations reports, plus a new DOD grant opportunity now open thanks to your advocacy.
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.
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.
Non-viral delivery gene editing takes an important step forward for Angelman syndrome
A FAST-funded study unveils STEP-RNP, a non-viral gene editing platform showing improved motor, cognitive function in AS mice—an early but promising step toward treating Angelman syndrome.
FAST LATAM Brings Families and Research Together in Mexico City
The second FAST LATAM Conference in Mexico City drew 267 in-person and nearly 496 virtual attendees, uniting families across the region around research updates and shared hope.
Using Vocalization Testing to Improve Detection of Treatment Effects in Angelman Syndrome Models
New research adds ultrasonic vocalization testing to Angelman syndrome mouse studies, offering a more sensitive way to detect communication-related changes and evaluate future treatments.