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Ultragenyx submits BLA to FDA for UX111

Ultragenyx submits BLA to FDA for UX111

Ultragenyx (RARE) Pharmaceutical announced the submission of a Biologics License Application, BLA, to the U.S. Food and Drug Administration seeking accelerated approval for UX111, ABO-102, AAV gene therapy as a treatment for patients with Sanfilippo syndrome type A

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