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3 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
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
University of California, San FranciscoNCT05619900
An Observational Disease and Clinical Outcomes Registry of Patients With Lysosomal Acid Lipase (LAL) Deficiency
This is an observational, multi-center, international disease registry designed to collect longitudinal data and create a knowledge base that will be utilized …
Lysosomal Acid Lipase DeficiencyCholesterol Ester Storage DiseaseWolman Disease+4 more
Alexion Pharmaceuticals, Inc.NCT01633489