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Ultragenyx Announces Positive Topline Cohort 2 Results from Phase 1/2 Clinical Study of DTX301 Gene Therapy in Ornithine Transcarbamylase (OTC) Deficiency and Progression to Higher Dose

Ultragenyx Pharmaceutical Inc.  (RARE) 
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PDF Ureagenesis normalized in one patient in Cohort 2 Cohort 1 responder demonstrated continued biochemical improvement and clinical stability at Week 52 Higher-dose Cohor [Read more]