Ultragenyx wins FDA approval for its Sanfilippo gene therapy
Ultragenyx ($RARE) received standard FDA approval for FAYUVI, the first approved treatment for Sanfilippo syndrome Type A. The single-infusion gene therapy treats neurological manifestations in pediatric patients with preserved neurodevelopmental function. The company expects commercial supplies to reach qualified US treatment centers within 30–60 days and also received a priority review voucher. The supporting program compared 17 treated patients with 27 patients from an external natural-histor
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