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