Status: Recruiting
Mucopolysaccharidosis VII Disease Monitoring Program
The objectives of this study are to characterize MPS VII disease presentation and progression and assess long-term effectiveness and safety, including hypersensitivity reactions and immunogenicity of vestronidase alfa.
Participants needed: 50
Trial details
Biological sex: AllType: ObservationalSponsor: Ultragenyx Pharmaceutical IncUpdated: Jun 11, 2026Locations: 14
Eligibility criteria
Diagnosis of MPS VII based on laboratory diagnosis, including either enzymatic o... [+2]
Concurrent participation in other pharmaceutical company-sponsored interventiona...