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Registry of Patients Diagnosed With Lysosomal Storage Diseases
This is an international prospective and retrospective registry of patients with Lysosomal Storage Diseases (LSDs) to understand the natural history of the dis…
Mucopolysaccharidosis IMucopolysaccharidosis IIMucopolysaccharidosis IV A+5 more
University of California, San FranciscoNCT05619900
Mucopolysaccharidosis VII Disease Monitoring Program (MPS VII DMP)
The objectives of this study are to characterize MPS VII disease presentation and progression and assess long-term effectiveness and safety, including hypersen…
Mucopolysaccharidosis VIIMPS VIIMPS 7+1 more
Ultragenyx Pharmaceutical IncNCT03604835