Ultragenyx Pharmaceutical Inc at Goldman Sachs Global Healthcare Conference (Virtual) Transcript
Good afternoon. I'm Salveen Richter, Biotechnology Analyst at Goldman Sachs, and we're pleased to have with us Ultragenyx with Emil Kakkis, CEO and Mardi Dier, CFO. Thanks for joining, Emil.
Questions & Answers
And maybe to start here, I was going to ask some big platform questions, but I might start with Angelman just given the news that, that hit.
So you reported this morning that the U.K. Medicines and Healthcare products Regulatory Agency approved the CTA for the Phase I/II study in pediatric patients. And you've also received clearance to enroll patients in Canada. And -- I believe, and you're expecting to enroll them in the second half of this year with clinical data for some patients by year-end. Maybe help us understand what this means to start and then we can walk into next steps?
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