Ultragenyx Pharmaceutical Inc at TD Cowen Healthcare Conference Transcript
(audio in progress) today, Eric Crombez, who's Chief Medical Officer and EVP at Ultragenyx. Eric, good to see you. Thanks for coming.
Thank you.
Questions & Answers
So lots going on in -- maybe we'll start with Angelman syndrome. That's going to be the next, I think, one of the big catalysts in the second half, maybe even Q3, the way we're kind of calculating and trying to back into a more fine-tuned timing. The Aspire study is about 130 patients, 4- to 17-year-old with a deletion. That's about 70% of patients fall into that, Randomized 1:1 versus sham. The primary endpoint is cognition based on the Bayley IV.
You obviously also have a Tandem study, the Aurora study, which we'll get into that in a second. When you're kind of thinking about powering for a benefit, what's considered clinically meaningful for
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