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Ultragenyx Pharmaceutical

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Ultragenyx Pharmaceutical’s $3.95 million FAYUVI gene therapy is the first FDA-approved disease-modifying treatment for children with Sanfilippo syndrome type A. Representative image.
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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Pharma & Biotech

What does September 19 UX111 PDUFA mean for Ultragenyx stock?

Ultragenyx faces a September 19 FDA decision for UX111 after RARE plunged on a separate trial failure and manufacturing issues delayed approval.

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FDA accelerated approval makes Genglycos the first treatment for glycogen storage disease type Ia, based on a 31% reduction in daily cornstarch intake, with long-term follow-up now needed to confirm the clinical benefit. Representative image.
Pharma & Biotech

Genglycos becomes first FDA-approved GSDIa therapy as Ultragenyx turns AAV8 gene therapy into a commercial product

FDA accelerated approval makes Genglycos the first treatment for GSDIa, based on a 31% reduction in daily cornstarch intake that must now be confirmed through long-term follow-up.

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