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Results For "Ultragenyx-Pharmaceutical"

2 News Found

Ultragenyx bags FDA priority review for groundbreaking GSDIa gene therapy
Drug Approval | February 24, 2026

Ultragenyx bags FDA priority review for groundbreaking GSDIa gene therapy

If approved, DTX401 would be the first treatment to address the disease at its root cause


Ultragenyx’s Setrusumab falls short in Phase 3 osteogenesis imperfecta trials
Clinical Trials | January 01, 2026

Ultragenyx’s Setrusumab falls short in Phase 3 osteogenesis imperfecta trials

The trials did not show a statistically significant reduction in annualized clinical fracture rates compared to placebo or bisphosphonates