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Sanfilippo syndrome

Pharma & Biotech

Why Ultragenyx stock jumped after FDA approved $3.95 million Fayuvi gene therapy

Ultragenyx Pharmaceutical has secured full FDA approval for Fayuvi, the first treatment for Sanfilippo syndrome Type A, giving the rare-disease biotech a major commercial catalyst only weeks after a Phase 3 setback erased nearly half its market value.

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