PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders) | ClinicalTrials HQ

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Trial	Conditions	Status	Phase	Updated	More info	NCT ID
PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)	Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Death, Death, Sudden, Gaucher Disease, Type 2, Gaucher Disease, Type 3, Gaucher disease, Infant Death, Infant, Newborn, Diseases, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Mucopolysaccharidosis Type IV A, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Skin and Connective Tissue Diseases, Sudden Infant Death, Wolman disease, brain disorder, central nervous system disorder, cholesteryl ester storage disease, connective tissue disorder, cutaneous mucinosis, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, mucopolysaccharidosis, mucopolysaccharidosis type 4, mucopolysaccharidosis type 6, mucopolysaccharidosis type 7, nervous system disorder, sphingolipidosis	Recruiting	Phase 1	2026-03-17		NCT04532047

2 results · Page 1 of 1

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Organizations	Linked trials
Duke University	446
University of California, San Francisco	637

1 results · Page 1 of 1

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Research sites	City	Country	Linked trials
University of California	San Francisco	United States	4