STAT | Reporting from the frontiers of health and medicine Follow STAT+: Ultragenyx drug to treat Angelman syndrome, a rare disease, fails late-stage trial Ultragenyx said its experimental therapy for Angelman syndrome, a rare disease, showed no benefit compared to a sham treatment in a Phase 3 trial. https://www.statnews.com/2026/09/02/ultragenyx-drug-angelman-syndrome-clinical-trial/ statnews.com
