Mucopolysaccharidosis Vii
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CLINICAL_TRIALS trial_result · 2026-04-07Clinical Trial Update: Expanded Access to Mepsevii
Expanded access program for Mepsevii (vestronidase alfa) for patients with mucopolysaccharidosis VII is currently available.
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CLINICAL_TRIALS trial_result · 2019-10-16Clinical 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]
Phase 2 clinical trial evaluating UX003 rhGUS enzyme replacement for MPS VII (Sly Syndrome) in children under 5; study completed with result…
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CLINICAL_TRIALS trial_result · 2019-07-30Clinical Trial Update: A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) [Phase 3]
Phase 3 trial evaluating UX003 rhGUS for MPS VII (Sly syndrome) has completed; results posted in 2019.