An expanded access record providing Mepsevii in MPS VII, Mucopolysaccharidosis VII and Sly Syndrome, sponsored by Ultragenyx Pharmaceutical Inc. Available. Per ClinicalTrials.gov, last updated 2026-07-30.
Sponsored by Ultragenyx Pharmaceutical Inc · Expanded access
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Ultragenyx Pharmaceutical Inc is the lead sponsor of 63 studies on the registry; 8 are open to participants now.
Of its 16 completed or terminated interventional studies of FDA-regulated products, 12 (75%) have results posted.
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Also known as: UX003, recombinant human beta-glucuronidase, rhGUS, vestronidase alfa
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Mucopolysaccharidosis VII
Ultragenyx Pharmaceutical Inc →