First Patient(s) Dosed in Ultragenyx Phase 3 Aspire Study

Ultragenyx Pharmaceutical Inc. announced today that the first patient(s) has/have been dosed in the pivotal Phase 3 Aspire study evaluating the efficacy and safety of GTX-102, its investigational antisense oligonucleotide (ASO) for Angelman syndrome.

Check clinicaltrials.gov for recruitment status and participation criteria.

Read the press release.

Watch the recent presentation by Ultragenyx at the 2024 FAST Global Science Summit.