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Ultragenyx Pharmaceutical
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Announcements
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Daily updates
- Ultragenyx received full FDA approval for FAYUVI in pediatric Sanfilippo syndrome Type ASep 17, 2026
- UX111 faced a September 19, 2026 PDUFA decision for Sanfilippo syndrome type ASep 7, 2026
- Ultragenyx Angelman syndrome trial missed its primary endpointSep 3, 2026
- FDA granted accelerated approval to GENGLYCOS for GSDIa patients aged 8 and olderAug 20, 2026
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News
- Ultragenyx received FDA approval for FAYUVI gene therapy, the first FDA-approved treatment for Sanfilippo syndrome type ASep 18, 2026
- Ultragenyx received U.S. FDA approval for GENGLYCOS, the first treatment designed to address the underlying cause of glycogen storage disease type IaAug 20, 2026
- Ultragenyx received FDA acceptance of BLA resubmission for UX111 gene therapy for Sanfilippo syndrome type AApr 2, 2026
- Ultragenyx received FDA IND clearance for UX016 to treat GNE myopathyMar 30, 2026
- Ultragenyx reported positive 36-week Phase 3 data for DTX301 AAV8 gene therapy in ornithine transcarbamylase deficiencyMar 12, 2026