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.
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.
Important update on alogabat, an investigational therapy in development for Angelman syndrome
Roche/Genentech has discontinued its alogabat program for Angelman syndrome. FAST thanks trial participants for their commitment and shares the full letter with additional context.
MavriX Bio MVX-220 Update
MavriX Bio’s gene therapy MVX-220 receives FDA Fast Track designation, with its Phase 1/2 ASCEND-AS trial now listed and a community webinar set for October 9.
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.