Ultragenyx Announces Upcoming Data Presentations at American Academy of Neurology (AAN) 2024 Annual Meeting

April 12, 2024

https://www.globenewswire.com/news-release/2024/04/12/2862138/20739/en/Ultragenyx-Announces-Upcoming-Data-Presentations-at-American-Academy-of-Neurology-AAN-2024-Annual-Meeting.html

NOVATO, Calif., April 12, 2024 (GLOBE NEWSWIRE) — Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) today announced that preclinical and clinical data, including new clinical efficacy and safety data from the expansion cohorts in the Phase 1/2 study of GTX-102 for the treatment of Angelman syndrome, will be presented at the 76th Annual American Academy of Neurology Meeting (AAN) being held April 13-18 in Denver and virtually. GTX-102 is an investigational antisense oligonucleotide delivered via intrathecal administration and is designed to target and inhibit expression of UBE3A antisense transcript (UBE3A-AS).

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