Clinical Trial Update: An Open-Label Phase 1/2 Study to Assess the Safety, Efficacy and Dose of Study Drug UX003 Recombinant Human Beta-glucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7) [Phase 1, Phase 2]

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Summary

Open-label Phase 1/2 trial assessing safety, efficacy, and dosing of UX003 rhGUS enzyme replacement in MPS VII has completed with results posted in 2018.

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Diseases / indications

• Mucopolysaccharidosis Type 7

Drugs mentioned

• UX003 Recombinant Human Beta-glucuronidase (recombinant human beta-glucuronidase)

Snippet

Sponsor: Ultragenyx Pharmaceutical Inc | Conditions: Mucopolysaccharidosis Type 7 | Phase 1, Phase 2 | Status: COMPLETED | Results posted: 2018-01-05