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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 PHARMACEUTICALS INC (RARE)  More Company Research Source: GlobeNewswire
Last ultragenyx pharmaceuticals inc earnings: 8/2 04:05 pm Check Earnings Report
US:NASDAQ Investor Relations: ir.ultragenyx.com/investor-relations
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, Calif., March 07, 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 positive longer-term safety and efficacy data from the first dose cohort of the Phase 1/2 study of DTX301, an investigational adeno-associated virus (AAV) gene therapy for the treatment of ornithine transcarbamylase (OTC) deficiency. “Longer term data from the first, lowest-dose cohort show that patient 1 maintained and substantially increased levels of ureagenesis through 24 weeks and we view these results as a promising indication of the potential and durability of DTX301. Based on these positive results at 24 weeks, this patient opted to discontinue all alternate pathway medication three weeks ago per t [Read more]

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