[ORGANIZATION]
Ultragenyx Pharmaceutical
Featured in 3 tracked stories · first seen
Situations
Silence Therapeutics financial and clinical developments
2 clusters · 3 sources · last updated
Latest: Silence Therapeutics stock rises 111 percent on successful clinical trial results
Silence Therapeutics, a biotechnology company specializing in RNA interference (RNAi) and siRNA therapies, has experienced significant financial and clinical developments. In August 2026, the company upsized a public of
FDA approval of Genglycos for GSDIa
2 clusters · 8 sources · last updated
Latest: FDA approves Genglycos for glycogen storage disease type Ia
The U.S. Food and Drug Administration (FDA) has granted accelerated approval to Ultragenyx Pharmaceutical’s gene therapy, Genglycos (pariglasgene brecaparvovec-opnr). This is the first authorized gene therapy for adult a
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Late-stage clinical trials fail to meet primary goals for rare disease treatments
[HEALTH] · 3 sources ·