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

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

~Estimated

~$10 – $5,625

University of California, San FranciscoNCT04532047