Sly Syndrome Completed Phase 3 Trials for Vestronidase alfa (DB12366)

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Also known as: Other mucopolysaccharidoses / Mucopolysaccharidosis VII / Mucopolysaccharidosis, MPS-VII (disorder) / Deficiency of beta-glucuronidase (disorder) / Sly's syndrome

IndicationStatusPhase
DBCOND0040268 (Sly Syndrome)Completed3
clinicaltrials.gov IdentifierTitlePurposeDrugs
NCT02230566
A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)Treatment
NCT02432144
A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7)Treatment