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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) 
Last ultragenyx pharmaceutical inc. earnings: 2/13 04:01 pm Check Earnings Report
US:NASDAQ Investor Relations: ir.ultragenyx.com/investor-relations
Company Research Source: GlobeNewswire
Ureagenesis normalized in one patient in Cohort 2 Cohort 1 responder demonstrated continued biochemical improvement and clinical stability at Week 52 Higher-dose Cohort 3 patient enrollment to begin in 2018; data expected in mid-2019 NOVATO, Calif., Sept. 27, 2018 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced topline positive safety and efficacy data from the second dose cohort and positive longer-term data from the first dose cohort of the ongoing Phase 1/2 study of DTX301, an investigational adeno-associated virus (AAV) gene therapy for the treatment of ornithine transcarbamylase (OTC) deficiency. “Data from the first two dose cohorts continue to demonstrate an acceptable initial safety profile and evidence of clinical activity, with one new patient achieving normalization of ureagenesis in Cohort 2 at 24 weeks,” said Eric Crombez, M.D Show less Read more
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