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Mucopolysaccharidosis Type VII - 10 Studies Found
Completed |
: 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 : Mucopolysaccharidosis Type 7 : 2013-05-08 : Drug: UX003 |
No longer available |
: An Open-Label Treatment Protocol With UX003 rhGUS Enzyme Replacement Therapy for an Advanced Stage MPS 7 Patient : Mucopolysaccharidosis Type 7 : 2014-03-24 : Drug: UX003 Open Label Other Names: |
Recruiting |
: Biomarker for Sly Disease :
|
Completed |
: A Phase 3 Study of UX003 rhGUS Enzyme Replacement Therapy in Patients With MPS 7 :
: |
Active, not recruiting |
: A Long-Term Open-Label Treatment and Extension Study of UX003 rhGUS Enzyme Replacement Therapy in Subjects With MPS 7 :
: Drug: UX003 UX003 is a sterile liquid buffered saline formulation of rhGUS that contains enzyme at a con |
Active, not recruiting |
: An Open-Label Study of UX003-rhGUS Enzyme Replacement Treatment in MPS 7 Patients Less Than 5 Years of Age :
: Drug: UX003 Other Names: |
Recruiting |
: Longitudinal Studies of Brain Structure and Function in MPS Disorders :
|
Terminated |
: Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism :
|
Recruiting |
: MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis :
|
Active, not recruiting |
: Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders :
: 2010-01-05 : |