3 studies found for: "Mucopolysaccharidosis type VII"
An Open-Label Phase 1/2 Study to Assess the Safety, Efficacy and Dose of Study Drug UX003 Recombinant Human Beta-glucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With MPS 7
Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders
Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism
† Indicates status has not been verified in more than two years