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0 -0.0007972665148063 -0.00227790432801809 -0.0153758542141229 -0.0125284738041002 -0.0153758542141229 -0.0150341685649202 -0.0258542141230068
Stock impact report

Ultragenyx (RARE) Meets Efficacy Goal in Neuro Disease Study [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
Per the data readout, 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 at day 170, which is either consistent with or exceeded the results from the Dose-escalation Cohorts 4-7 data. Such improvements across multiple functional domains include improvements in cognitive function, behavior, hyperactivity and noncompliance, sleep, receptive communication and gross motor function. Ultragenyx further reported that the treatment of Angelman syndrome patients in the Dose-escalation Cohorts 4-7 with GTX-102 demonstrated long-term increasing and sustained clinical benefit far exceeding the Natural History study data of the candidate at day 758. Despite the positive results, the stock lost 8.8% in the last trading session on Apr 15. The decline in the share price was likely due to reports of three patients in the phase I/II study of GTX-102 who experienced serious adverse events (m Show less Read more
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