An Ultragenyx gene therapy has scored a win at the midway point of its phase 3 trial, successfully reducing ammonia levels in patients with a rare urea cycle disorder.
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- Ultragenyx gene therapy hits co-primary endpoint in phase 3 rare disease study, teeing up potential win
Ultragenyx gene therapy hits co-primary endpoint in phase 3 rare disease study, teeing up potential win
- March 12, 2026
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