Browsing Tag
FAYUVI
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Ultragenyx adds sixth FDA-approved product as FAYUVI targets Sanfilippo syndrome
Ultragenyx Pharmaceutical Inc. has secured full FDA approval for FAYUVI, a $3.95 million one-time gene therapy for Sanfilippo syndrome type A, giving the rare-disease company a major commercial catalyst weeks after another program wiped 44% from its share price.
September 24, 2026