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Showing 1-2 of 2 trials for Mps-vi
Recruiting

PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)

California · San Francisco, CA

For detailed information, please view our study website: https://pearltrial.ucsf.edu/ The investigators aims to determine the the maternal and fetal safety and feasibility of in utero fetal enzyme replacement therapy in fetuses with Lysosomal Storage Diseases.

Recruiting

Mucopolysaccharidosis VII Disease Monitoring Program

California · Orange, CA

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.