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Ultragenyx's DTX301 Hits Primary Endpoint in Phase 3 OTC Deficiency Trial — But First-Generation AAV Faces Biological and Competitive Limits
Enh3ance Study Delivers Statistically Significant Ammonia Reduction and Scavenger Tapering Versus Placebo, Yet Neonatal Exclusion, Seroprevalence Barriers, and Advancing mRNA and Gene Editing Rivals Define the Ceiling for a Curative Therapy. See Disclaimer below * For the estimated 10,000 people living with Ornithine Transcarbamylase (OTC) deficiency in commercially accessible geographies,