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Stock impact report

Ultragenyx Announces Positive Interim Phase 1/2 Data in Patients with Angelman Syndrome After Treatment with GTX-102 [Yahoo! Finance]

Ultragenyx Pharmaceutical Inc. (RARE) 
Last ultragenyx pharmaceutical inc. earnings: 2/13 04:01 pm Check Earnings Report
US:NASDAQ Investor Relations: ir.ultragenyx.com/investor-relations
Company Research Source: Yahoo! Finance
Additional long-term data in Dose-escalation Cohorts showed increasing and sustained clinical benefit through Day 758 Company will host investor call at 8:00 a.m. ET NOVATO, Calif., April 15, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced new data from the Phase 1/2 study of GTX-102 for the treatment of Angelman syndrome. Patients in Expansion Cohorts A & B treated with a set dose and regimen of GTX-102 showed rapid and clinically meaningful improvement across multiple domains consistent with or exceeding Dose-escalation Cohorts 4-7 data at Day 170. Treatment of the Dose-escalation Cohorts 4-7 showed long-term increasing and sustained clinical benefit far exceeding Natural History data at Day 758. These data will be discussed in more detail in a corporate presentation being hosted by the company today at 8:00 a.m. ET and will also be presented by Kemi Olugemo, M.D., FAAN at the 76th Annual American Academy of Neurology Meeting (AAN) in Denv Show less Read more
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