Clinical trials: MPS 7, open to volunteers
Mucopolysaccharidosis VII, MPS VII
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.
- Conditions
- Mucopolysaccharidosis VII, MPS VII, MPS 7, Sly Syndrome
- Trial name
- NCT03604835
- Testing
- No Intervention
- Where
- 14 sites in United States, Argentina and 6 more
- Volunteers
- 50 people
- Sponsor
- Ultragenyx Pharmaceutical Inc