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Found 8 studies with search of: | "niemann-pick disease" |
Rank | Status | Study | ||||
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1 | Terminated |
Safety Study of rhASM Enzyme Replacement Therapy in Adults With Acid Sphingomyelinase Deficiency (Niemann-Pick Disease)
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2 | Recruiting |
Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism
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3 | Recruiting |
Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type C
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4 | Active, not recruiting |
Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation
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5 | Completed |
Stem Cell Transplant for Inborn Errors of Metabolism
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6 | Recruiting |
Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells
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7 | Completed |
PET Scan of Brain Metabolism in Relation to Age and Disease
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8 | Completed |
Saccadic Eye Movements in Patients With Niemann-Pick Type C Disease
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