Ultragenyx Pharmaceutical Inc at Goldman Sachs Global Healthcare Conference Transcript
Good afternoon. Thank you, everyone, for joining us. Really pleased to have the Ultragenyx team here with us. We have Emil Kakkis, Founder, President and CEO; and Mardi Dier, CFO.
Questions & Answers
With that, Emil, data sets that are going to start to appear in the second half onwards, let's start with the Angelman program. As we look to that midyear efficacy and safety data update from the Phase I/II study, can you remind us of the metrics that will be considered, the number of patients we'll see data on? And what you would interpret as a positive signal for the program?
Sure. So we'll be releasing data on Cohort 4, which is less than 8 group; and Cohort 5, probably the majority of patients. We will have -- the hope is to get the majority of patient Day 128 data. And what
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