5 studies found for: "mucopolysaccharidosis type VII"
MT2013-31:Allo BMT for Metabolic Disorders, Osteopetrosis and Males With Rett Syndrome
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
|3||Temporarily not available||
An Open-Label Treatment Protocol With UX003 rhGUS Enzyme Replacement Therapy for an Advanced Stage MPS 7 Patient
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