Ultragenyx Announces Positive 36-Week Data from Phase 3 Study of DTX301 AAV8 Gene Therapy for the Treatment of Ornithine Transcarbamylase (OTC) Deficiency

Statistically significant improvements in primary endpoint of ammonia control compared with placebo at 36 weeks Clinically important changes observed in patient global impression scale for overall OTC symptoms, OTC deficiency symptoms, and OTC impact on daily living DTX301 was well tolerated with