Angelman syndrome drug GTX-102 misses Phase 3 endpoint, deal setback for Ultragenyx
TL;DR Summary
Ultragenyx said its experimental Angelman syndrome therapy GTX-102 showed no benefit versus a sham in a large Phase 3 trial, ending earlier hopes from earlier results and creating a major hurdle for the company's profitability plans in the rare-disease space.
- Ultragenyx drug to treat Angelman syndrome, a rare disease, fails late-stage trial STAT
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