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

~Estimated

~$10 – $1,050

Ultragenyx Pharmaceutical IncNCT03604835

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

~Estimated

~$10 – $675

University of California, San FranciscoNCT05619900