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Ultragenyx启动基因疗法的FDA营销申请程序

2025-08-18 21:02

Ultragenyx Pharmaceutical (NASDAQ:RARE) said that it has started the rolling submission of a marketing application to the U.S. FDA for its DTX401 AAV gene therapy, aimed at treating glycogen storage disease Type Ia.
The company has already provided the FDA with the non-clinical and clinical sections of the application.
They plan to finalize the completion of the application by the end of 2025, which will include details on chemistry, manufacturing, and controls.

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