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.
Ionis Pharmaceuticals: A letter to the Angelman syndrome community.
Ionis addresses community questions following the ASPIRE trial results, reaffirming its commitment to obudanersen (ION582) through the HALOS, REVEAL, and CHAMPION trials
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.
Ultragenyx Announces Phase 3 Aspire results in Angelman Syndrome
The Phase 3 ASPIRE study for GTX-102 (apazunersen) did not meet its primary or key secondary endpoints. FAST expresses gratitude to participating families and awaits further details from Ultragenyx.
The Spark That Brought a Community Together
The first-ever Chloe’s Spark Golf Scramble in Minnesota raised over $55,000, bringing together family and friends to celebrate Chloe’s joy and support Angelman syndrome research.
From Backyard Cornhole to Big Impact: For the Love of Linc
In its first year, the Ciotola family’s cornhole tournament, For the Love of Linc, raised more than $8,000 for Angelman syndrome, turning backyard fun into community impact.
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.
The LaMuras organized Rally for a Cure, a Tennis-Themed Event that Brought Together Their Community
Monica LaMura’s first-ever fundraiser, Rally for a Cure, brought nearly 50 kids together for a tennis-themed event, raising $5,000 for FAST in honor of her son Colin.
Charlie’s Angels Chari-Tee Golf Tournament: A Day of Hope, Community, and Action
In its 5th year, Charlie’s Angels Chari-Tee Golf Tournament raised over $90,000 in a single day, bringing its total to nearly $300,000 for Angelman syndrome research.
Nobel Laureate Frances Arnold Brings Directed Evolution to Angelman Syndrome Research
Nobel laureate Dr. Frances Arnold receives FAST funding to engineer new molecular tools for durable RNA-targeted therapies, bringing world-class expertise to Angelman syndrome research.