10 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
|2||No longer available||
An Open-Label Treatment Protocol With UX003 rhGUS Enzyme Replacement Therapy for an Advanced Stage MPS 7 Patient
Biomarker for Sly Disease
A Phase 3 Study of UX003 rhGUS Enzyme Replacement Therapy in Patients With MPS 7
A Long-Term Open-Label Treatment and Extension Study of UX003 rhGUS Enzyme Replacement Therapy in Subjects With MPS 7
|6||Active, not recruiting||
An Open-Label Study of UX003-rhGUS Enzyme Replacement Treatment in MPS 7 Patients Less Than 5 Years of Age
Longitudinal Studies of Brain Structure and Function in MPS Disorders
Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism
MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
|10||Active, not recruiting||
Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders
† Study has passed its completion date and status has not been verified in more than two years.