Clinical Trial Update: Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of Age [Phase 2]

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Summary

Phase 2 clinical trial evaluating UX003 rhGUS enzyme replacement for MPS VII (Sly Syndrome) in children under 5; study completed with results posted in 2019.

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Diseases / indications

• Mucopolysaccharidosis Vii
• Sly Syndrome

Drugs mentioned

• UX003 (recombinant human beta-glucuronidase)

Snippet

Sponsor: Ultragenyx Pharmaceutical Inc | Conditions: Sly Syndrome, MPS VII | Phase 2 | Status: COMPLETED | Results posted: 2019-10-16