Ultragenyx completes rolling submission of BLA for DTX401
US biotech Ultragenyx Pharmaceutical In (Nasdaq: RARE) this week revealed that it has completed the rolling submission of its Biologics License Application (BLA) to the US Food and Drug Administration (FDA), seeking approval for DTX401 AAV gene therapy (pariglasgene brecaparvovec) as a treatment for glycogen storage disease Type Ia (GSDIa).
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