Gene Therapy with Modified Autologous Hematopoietic Stem Cells for Patients with Mucopolysaccharidosis Type IIIA | ClinicalTrials HQ

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Trial	Conditions	Status	Phase	Updated	More info	NCT ID
Gene Therapy with Modified Autologous Hematopoietic Stem Cells for Patients with Mucopolysaccharidosis Type IIIA	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, mucopolysaccharidosis type 3A	Active, not recruiting	Phase 1 / Phase 2	2025-03-30		NCT04201405

6 results · Page 1 of 1

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Organizations	Linked trials
CTI Clinical Trial and Consulting Services	4
Great Ormond Street Hospital for Children NHS Foundation Trust	34
Manchester University NHS Foundation Trust	41
Orchard Therapeutics	5
University College, London	222
University of Manchester	85

1 results · Page 1 of 1

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Research sites	City	Country	Linked trials
Manchester University NHS Foundation Trust	Manchester	United Kingdom	22