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Sanfilippo syndrome

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FDA Accepts Ultragenyx's Gene Therapy Application for Sanfilippo Syndrome, Setting September Decision Date for First-Ever Treatment

The FDA has accepted Ultragenyx's resubmitted BLA for UX111, a one-time gene therapy for the fatal childhood disease Sanfilippo syndrome Type A, with a decision expected by September 19, 2026.

5 min read3 sources