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FAST Angelman Syndrome News

  • Industry

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

  • Fundraising

For Jack: Sonia and Jose Take on the Inca Trail

Before Sonia and her son José set off on Peru’s Inca Trail this September, their guides gave the group two goals: survive first, and finish if you can. For Sonia

  • Industry

MavriX Bio Announces FDA Rare Pediatric Disease Designation for Investigational MVX-220 for the Treatment of Angelman Syndrome

Read the press release

  • Research

Beyond UBE3A: New Conversations About Genotype, Variants, and Future Treatments

At INSYNC-AS 2026, researchers came together to examine how genes beyond UBE3A and differences within UBE3A itself may shape Angelman syndrome and future therapeutic development. Why can Angelman syndrome (AS)

  • Industry

Skylark Bio Expands Pipeline with First CNS Development Program, SKY-AS, Targeting UBE3A Restoration in Angelman Syndrome

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  • Industry

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.
  • Industry

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

  • Industry

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
  • Industry

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

  • Industry

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.
  • Industry

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.
  • Fundraising

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.
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