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Ultragenyx Announces Positive Topline Results and DMC Review from First Cohort of Phase 1/2 Clinical Study of DTX301, an Investigational Gene Therapy in Ornithine Transcarbamylase (OTC) De...

Ultragenyx Pharmaceutical Inc.  (RARE) 
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PDF Ureagenesis normalized in one patient and further increased by 24 weeks Cohort 2 patient enrollment to begin in March 2018; data expected in second half of 2018 NOVATO [Read more]