Browsing Tag
Sanfilippo Syndrome Type A
1 post
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.