Ultragenyx Pharmaceutical (NASDAQ:RARE) is up 10% premarket on light volume in reaction to positive data from the second cohort in its Phase 1/2 clinical trial evaluating gene therapy DTX401 in patients with glycogen storage disease type Ia. The data were presented at SSIEM in Rotterdam.
All three patients responded with improvements in glucose control and other metabolic measures compared to baseline.
Participants in the first cohort (lower dose) continue to experience sustained responses.
The estimated completion date is September 2020.
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