Leukodystrophy - 153 Studies Found
Recruiting |
: UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells :
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Completed |
: Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation :
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Recruiting |
: MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis :
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Completed |
: Stem Cell Transplant for Inborn Errors of Metabolism :
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Active, not recruiting |
: The Natural History of Metachromatic Leukodystrophy : Metachromatic Leukodystrophy : 2008-03-11 |
Recruiting |
: Intracerebral Gene Therapy for Children With Early Onset Forms of Metachromatic Leukodystrophy : Metachromatic Leukodystrophy : 2013-01-28 : Genetic: intracerebral administration of AAVrh.10cuARSA |
Completed |
: Multicenter Study of HGT-1110 Administered Intrathecally in Children With Metachromatic Leukodystrophy (MLD) : Metachromatic Leukodystrophy (MLD) : 2011-12-14 : Biological: Recombinant human arylsulfatase A 6 patients treated with HGT-1110 EOW by IT injection |
Completed |
: Effect of Warfarin in the Treatment of Metachromatic Leukodystrophy : Metachromatic Leukodystrophy : 2008-05-21 : Drug: Warfarin Oral administration (QD), variable dosage: patients will undergo blood test for PT/INR at |
Withdrawn |
: An Observational Study of Pediatric Subjects With Globoid Cell Leukodystrophy (GLD) : Leukodystrophy, Globoid Cell : 2010-03-24 |