Ultragenyx reports positive Phase 1/2 results for GTX-102 in Angelman syndrome, showing improvements in cognition, communication, behavior, and sleep.

Ultragenyx Pharmaceutical Inc. announced positive Phase 1/2 study results for GTX-102 in Angelman syndrome, showing rapid and meaningful improvements in cognition, communication, behavior, and sleep. Despite three related adverse events, the data exceeded natural history data and the company plans to engage with authorities for a Phase 3 pivotal study. Currently, no disease-modifying treatment is approved for Angelman syndrome.