Washington University School of Medicine Division of Genetics and Genomic Medicine, Rare Diseases Department of Pediatrics

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Trial	Conditions	Status	Phase	Updated	More info	NCT ID
Single Injection of rAAV9-CMV-hNAGLUop Gene for Patients With Mucopolysaccharidosis (MPS) IIIB	Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Mucopolysaccharidosis III, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, connective tissue disorder, cutaneous mucinosis, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, lysosomal storage disease, metabolic disease, mucopolysaccharidosis	Not yet recruiting	Phase 1 / Phase 2	2026-09-14		NCT07818759

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