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2 recruiting studies matching your filters
Phase 1
PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)
For detailed information, please view our study website: https://pearltrial.ucsf.edu/ The investigators aims to determine the the maternal and fetal safety an…
MPS IMPS IIMPS IVA+6 more
University of California, San FranciscoNCT04532047
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