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 endpoints. The webinar recording is now available: https://youtu.be/Jl43UtOVFm4
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.
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
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.
Ultragenyx Completes Enrollment of Phase 3 Aspire Study Evaluating GTX-102 for the Treatment of Angelman Syndrome
Ultragenyx announces the Phase 3 ASPIRE study for apazunersen (GTX-102) is fully enrolled with 129 participants, reaching this milestone in just seven months.
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.
Ultragenyx Announces First Patient(s) Dosed in Pivotal Phase 3 Aspire Study Evaluating GTX-102 in Angelman Syndrome
Ultragenyx announces the first patients dosed in its pivotal Phase 3 ASPIRE study evaluating GTX-102 for Angelman syndrome, a major step toward potential treatment.
Ultragenyx Community Update October 2024
The Phase 3 Efficacy and Safety Study of GTX-102 (ASO) in Pediatrics, sponsored by Ultragenyx, is now listed on ClinicalTrials.gov. Interested in learning more? Watch the Ultragenyx Community Webinar
Ultragenyx Announces Successful End-of-Phase 2 Meeting with FDA for GTX-102 Angelman Syndrome Program
Today, Ultragenyx Pharmaceuticals shared a press release announcing a “Successful End-of-Phase 2 Meeting with FDA for GTX-102 Angelman Syndrome Program.” Read the full press release here.
Ultragenyx Announces Positive Interim Phase 1/2 Data in Patients with Angelman Syndrome After Treatment with GTX-102
We’re thrilled to share an update from Ultragenyx regarding reported positive interim data from the ongoing Phase 1/2 study in patients living with deletion genotype Angelman syndrome after treatment with investigational GTX-102. From company press release: “The totality of these interim data demonstrates that treatment with GTX-102 resulted in rapid, multi-domain improvements that continued during […]