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Clinical Trials for Mucopolysaccharidosis VII

  • A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7)
  • Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of Age
  • A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)
  • MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
  • Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders
  • Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism
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