Ultragenyx Receives Orphan Drug Designation from FDA and European Commission for UX053 ...

Retrieved on: 2021-07-27 12:00:00

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Health

Life Sciences

Drug discovery

Hepatology

Inborn errors of carbohydrate metabolism

Food and Drug Administration

Ultragenyx

Pharmaceuticals policy

Arcturus Therapeutics

Orphan drug

Glycogen storage disease type II

Clinical trial

Ultragenyx Pharmaceutical Inc

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Ultragenyx Receives Orphan Drug Designation from FDA and European Commission for UX053 .... View article details on hiswai:

Excerpt

GSDIII affects more than 10,000 patients in the developed world. About UX053. UX053 is an investigational mRNA-based biologic therapy encoding full- ...

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